Pipeline Watchlist

Company Pipelines

Recent additions

14 days
CYBN
Cybin Inc.

HLP003

Mental health / Phase 3 / Adjunctive treatment of Major Depressive Disorder (MDD)

helus.com
CVAC
CureVac N.V.

UPEC FimH ferritin nanoparticle mRNA vaccine candidate

Infectious disease / Preclinical / urinary tract infections caused by uropathogenic E. coli

curevac.com
CVAC
CureVac N.V.

COVID-19/influenza combination mRNA vaccine candidate

Infectious disease / Phase 1/2 / COVID-19 and influenza

curevac.com
CVAC
CureVac N.V.

sqNSCLC shared-antigen cancer precision immunotherapy candidate

Oncology / IND/CTA filed / squamous non-small cell lung cancer

curevac.com

Companies

62assets
  • LutatheraAAA601 / Lutathera / SSTR / Radioligand therapy / Oncology: Solid Tumors
    Gastroenteropancreatic neuroendocrine tumorsPhase 3Supplementary; filing 2028GEPNET, pediatricsPhase 2
  • 177Lu-NeoBAAA603 / GRPR / Radioligand therapy / Oncology: Solid Tumors
    Breast cancerPhase 1Multiple solid tumorsPhase 2Lead; filing >= 2029
  • AAA614FAP / Radioligand therapy / Oncology: Solid Tumors
    Solid tumorsPhase 2Lead
  • PluvictoAAA617 / Pluvicto / PSMA / Radioligand therapy / Oncology: Solid Tumors
    Oligometastatic prostate cancerPhase 3Supplementary; filing >= 2029Metastatic hormone sensitive prostate cancer (mHSPC)RegistrationSupplementary
  • 225Ac-PSMA-617AAA817 / PSMA / Radioligand therapy / Oncology: Solid Tumors
    Metastatic castration-resistant prostate cancer (mCRPC)Phase 3Supplementary; filing >= 2029post Lu Metastatic castration-resistant prostate cancer (mCRPC)Phase 3Lead; filing 2028
  • ScemblixABL001 / Scemblix / BCR-ABL / Inhibitor / Oncology: Hematology
    Chronic myeloid leukemia, pediatricsPhase 2Supplementary; filing 2027
  • CosentyxAIN457 / Cosentyx / IL17A / Inhibitor / Immunology
    Polymyalgia rheumaticaRegistrationSupplementary; filing 2026
  • AimovigAMG334 / Aimovig / CGRPR / Antagonist / In-market Brands and Global Health
    Migraine, pediatricsPhase 3Supplementary; filing 2028
  • AMO959DNA PK / Inhibitor / Oncology: Solid Tumors
    Prostate cancerPhase 1Lead
  • MayzentBAF312 / Mayzent / S1P1,5 receptor modulator / Neuroscience
    Multiple sclerosis, pediatricsPhase 3Supplementary; filing 2027
  • VijoyceBYL719 / Vijoyce / PI3K-alpha / Inhibitor / Oncology: Solid Tumors
    Lymphatic malformationsPhase 3Supplementary; filing >= 2029
  • farabursenCYX082 / MIR17 / Inhibitor / Cardiovascular, Renal and Metabolic
    Autosomal dominant polycystic kidney diseasePhase 1Lead
  • pelabresibDAK539 / BET / Inhibitor / Oncology: Hematology
    MyelofibrosisPhase 3Lead; filing 2026
  • DFT383CTNS gene delivery / Neuroscience
    CystinosisPhase 1Lead
  • DII235siRNA targeting Lp(a) MRNA / Cardiovascular, Renal and Metabolic
    CVRR-Lp(a)Phase 2Lead; filing >= 2029
  • del-braxDWH213 / siRNA DUX4 knockdown / Neuroscience
    Facioscapulohumeral muscular dystrophyPhase 3Lead; filing 2028
  • tulmimetostatDZR123 / EZH1, EZH2 / Inhibitor / Oncology: Solid Tumors
    Metastatic prostate cancerPhase 1Solid tumors & lymphomasPhase 2Lead
  • ECI830CDK2 / Inhibitor / Oncology: Solid Tumors
    Breast cancerPhase 1Lead
  • EDI048CpPI(4)K / Inhibitor / In-market Brands and Global Health
    CryptosporidiosisPhase 2Lead
  • EDK060Lead Indication / Neuroscience
    Charcot-Marie-Tooth diseasePhase 1
  • ESP359DLL3 / Radioligand therapy / Oncology: Solid Tumors
    Solid tumorsPhase 1Lead
  • del-desiranEWF980 / siRNA DMPK knockdown / Neuroscience
    Myotonic dystrophy type 1Phase 3Lead; filing 2027
  • EYU688NS4B / Inhibitor / In-market Brands and Global Health
    Dengue feverPhase 2Lead
  • zigakibartFUB523 / Anti-APRIL / Cardiovascular, Renal and Metabolic
    IgA nephropathyPhase 3Lead; filing 2027
  • 177Lu-NNS309FXX489 / FAP / Radioligand therapy / Oncology: Solid Tumors
    Solid tumorsPhase 1Lead
  • GCJ904Lead Indication / Oncology: Solid Tumors
    Solid tumorsPhase 1
  • GHZ339Lead Indication / Immunology
    Atopic dermatitisPhase 2filing >= 2029
  • GIA632IL-15 mAb / Immunology
    Atopic dermatitisPhase 2Lead; filing >= 2029VitiligoPhase 2Supplementary; filing >= 2029
  • GXV813M4 / Agonist / Neuroscience
    SchizophreniaPhase 2Lead
  • HJB647Lead Indication / Cardiovascular, Renal and Metabolic
    Heart failurePhase 1
  • votoplamHTT227 / Huntingtin modulator / Neuroscience
    Huntington’s diseasePhase 3Lead; filing >= 2029
  • INE963Plasmodium falciparum / Inhibitor / In-market Brands and Global Health
    MalariaPhase 2Lead
  • IPX643Lead Indication / Immunology
    Inflammation-driven diseasesPhase 1
  • ITU512HbF inducing agent / In-market Brands and Global Health
    Sickle cell diseasePhase 1Lead
  • luxdegalutamideJSB462 / Androgen receptor protein degrader / Oncology: Solid Tumors
    Metastatic hormonal sensitive prostate cancerPhase 2Metastatic castration resistant prostate cancerPhase 2Lead; filing >= 2029
  • cipargaminKAE609 / PfATP4 / Inhibitor / In-market Brands and Global Health
    Malaria, uncomplicatedPhase 2Supplementary; filing >= 2029Malaria severePhase 2Lead; filing >= 2029
  • LeqvioKJX839 / Leqvio / siRNA (regulation of LDL-C) / Cardiovascular, Renal and Metabolic
    CVRR (primary prevention)Phase 3Supplementary; filing >= 2029CVRR (secondary prevention)Phase 3Supplementary; filing 2027
  • Ganaplacide + lumefantrineKLU156 / Non-artemisinin plasmodium falciparum / Inhibitor / In-market Brands and Global Health
    Malaria, uncomplicatedPhase 3Lead; filing 2026
  • del-zotaKPE179 / PMO mediated exon 44 skipping / Neuroscience
    Duchenne muscular dystrophyPhase 2Lead; filing 2026
  • FabhaltaLNP023 / Fabhalta / CFB / Inhibitor / Neuroscience / Cardiovascular, Renal and Metabolic / Oncology: Hematology / Others
    Generalized myasthenia gravisPhase 3Supplementary; filing 2027C3 glomerulopathy, pediatricsPhase 3IgAN, pediatricsPhase 3
  • RhapsidoLOU064 / Rhapsido / BTK / Inhibitor / Immunology
    Chronic spontaneous urticaria, pediatricsPhase 3Hidradenitis suppurativaPhase 3Supplementary; filing 2027Chronic inducible urticaria (CINDU)Phase 3Supplementary; filing 2026
  • remibrutinibLOU064 / BTK / Inhibitor / Neuroscience
    Multiple sclerosis, secondary progressivePhase 3Supplementary; filing >= 2029Myasthenia gravisPhase 3Supplementary; filing 2028Multiple sclerosis, relapsing remittingPhase 3Supplementary; filing 2027
  • LTP001SMURF1 / Inhibitor / Cardiovascular, Renal and Metabolic
    Idiopathic pulmonary fibrosisPhase 2Pulmonary arterial hypertensionPhase 2Lead; filing >= 2029
  • LXE408Proteasome / Inhibitor / In-market Brands and Global Health
    ChagasPhase 2Visceral leishmaniosisPhase 2Lead; filing >= 2029
  • abelacimabMAA868 / FXI / Inhibitor / Cardiovascular, Renal and Metabolic
    Atrial fibrillationPhase 3Lead; filing 2028
  • MAS825IL18 Inhibitor, IL1B / Immunology
    Still’s diseasePhase 2Lead
  • NIO752Tau antisense oligonucleotide / Neuroscience
    Alzheimer’s diseasePhase 1Progressive supranuclear palsyPhase 1Lead
  • OJR520Lead Indication / Cardiovascular, Renal and Metabolic
    CKDPhase 1
  • KesimptaOMB157 / Kesimpta / CD20 / Antagonist / Neuroscience
    Multiple sclerosis, new dosing regimenPhase 3Supplementary; filing 2027Multiple sclerosis, pediatricsPhase 3Supplementary; filing 2026
  • PAC001Anti-IL-6 mAb / Cardiovascular, Renal and Metabolic / Others
    ASCVD pacibekitugPhase 2Lead; filing >= 2029Thyroid eye disease (TED) pacibekitugPhase 2
  • PIT565Anti-CD19, Anti-CD2, Anti-CD3 / Immunology
    Systemic lupus erythematosusPhase 1Rheumatoid arthritisPhase 1
  • RydaptPKC412 / Rydapt / Multi-targeted kinase / Inhibitor / In-market Brands and Global Health
    Acute myeloid leukemia, pediatricsPhase 2Supplementary; filing 2027
  • PKN605HDAC6 / Inhibitor / Cardiovascular, Renal and Metabolic
    Atrial FibrillationPhase 2Lead
  • QCZ484Lead Indication / Cardiovascular, Renal and Metabolic
    HypertensionPhase 2filing >= 2029
  • AtecturaQMF149 / Atectura / LABA + ICS / In-market Brands and Global Health
    Asthma, pediatricsPhase 3Supplementary; filing >= 2029
  • AdakveoSEG101 / Adakveo / P-selectin / Inhibitor / In-market Brands and Global Health
    Sickle cell disease, pediatricsPhase 3Supplementary; filing >= 2029
  • TIN816ATP modulator / Cardiovascular, Renal and Metabolic
    Acute kidney injuryPhase 2Lead
  • pelacarsenTQJ230 / ASO targeting Lp(a) / Cardiovascular, Renal and Metabolic
    CVRR (secondary prevention) in patients with elevated Lp(a)Phase 3Lead; filing 2026
  • ianalumabVAY736 / BAFF-R inhibitor, ADCC-mediated B-cell depletor / Immunology / Oncology: Hematology
    Lupus NephritisPhase 3Supplementary; filing 2028Systemic lupus erythematosus (SLE)Phase 3Supplementary; filing 2028Systemic sclerosisPhase 2Supplementary; filing 2028
  • VHB937TREM2 stabilizer and activator / Neuroscience
    Amyotrophic lateral sclerosisPhase 2Lead; filing >= 2029Alzheimer's diseasePhase 2Supplementary; filing >= 2029
  • YMI024Lead Indication / Cardiovascular, Renal and Metabolic
    CVRRPhase 1
  • rapcabtagene autoleucelYTB323 / CD19 CAR-T / Immunology / Neuroscience / Oncology: Hematology
    Rheumatoid arthritis and severe, refractory Sjögren’s diseasePhase 1Systemic sclerosisPhase 2Supplementary; filing >= 2029MyositisPhase 2Supplementary; filing >= 2029
37assets
  • ABBV-8736ABBV-8736 / TREM1 / Biologic / Immunology
    Crohn's DiseasePhase 2
  • TavapadonD1/D5 partial agonist / Small molecule / Neuroscience
    Parkinson's DiseaseSubmitted
  • ABBV-525ABBV-525 / MALT1 / Small molecule / Oncology
    Hematologic TumorsPhase 1
  • Telisotuzumab vedotinABBV-399 / EMRELIS / c-Met / Antibody-drug conjugate / Oncology
    Non-Small Cell Lung CancerApprovedUS
  • EpcoritamabGen3013 / EPKINLY/TEPKINLY / CD3, CD20 / Bispecific antibody / Oncology
    Relapsed/Refractory DLBCLApprovedUSEURelapsed/Refractory Follicular LymphomaApprovedRelapsed/Refractory CLLPhase 2
  • ABBV-466 (Skyrizi + ABBV-382)ABBV-466 / SKYRIZI / IL-23 + anti-a4b7 mAb / Biologic combination / Immunology
    Crohn's DiseasePhase 2
  • BudigalimabABBV-181 / PD1 / Biologic / Oncology
    Solid and Hematologic TumorsPhase 2
  • ABBV-701ABBV-701 / TL1A / Biologic / Immunology
    Inflammatory Bowel DiseasePhase 1
  • ABBV-932ABBV-932 / DRD2/DRD3 modulator / Small molecule / Neuroscience
    Bipolar DepressionPhase 2Generalized Anxiety DisorderPhase 2
  • ABBV-9832 (Lutikizumab + Ravagalimab)ABBV-9832 / IL-1a/1b + CD40 antagonist / Biologic combination / Immunology
    Rheumatoid ArthritisPhase 2
  • ABBV-6628ABBV-6628 / C5 / Biologic / Eye Care
    Geographic AtrophyPhase 1
  • ABBV-619ABBV-619 / CD19 / Biologic/CAR-T / Immunology
    Autoimmune diseasesPhase 1
  • LivmoniplimabABBV-151 / GARP-TGF-beta1 / Biologic / Oncology
    Hepatocellular CarcinomaPhase 2Solid TumorsPhase 2Non-Small Cell Lung CancerPhase 2
  • Armour ThyroidArmour Thyroid / T3T4 / Biologic / Other Specialties
    HypothyroidismPhase 3
  • ABBV-319ABBV-319 / CD19 / Biologic ADC / Immunology
    Sjogren's diseasePhase 1Systemic Lupus ErythematosusPhase 1
  • ABBV-722ABBV-722 / LPAR1 / Small molecule / Immunology
    Idiopathic Pulmonary FibrosisPhase 1Systemic SclerosisPhase 1
  • ABBV-142ABBV-142 / LPAR1 / Biologic / Immunology
    Idiopathic Pulmonary FibrosisPhase 1
  • ABBV-324ABBV-324 / Biologic ADC / Oncology
    Solid TumorsPhase 1
  • ABBV-295ABBV-295 / Long-Acting Amylin Analog / Large molecule / Other Specialties
    ObesityPhase 1
  • SurzetoclaxABBV-453 / BCL-2 / Small molecule / Oncology
    Relapsed/Refractory Multiple MyelomaPhase 1
  • ABBV-2002ABBV-2002 / PAR-2 / Biologic / Neuroscience
    MigrainePhase 2
  • IcalcaprantABBV-1354 / KOR antagonist / Small molecule / Neuroscience
    Bipolar DepressionPhase 2Major Depressive DisorderPhase 2
  • ABBV-1042ABBV-1042 / M4 receptor / Small molecule / Neuroscience
    Bipolar ManiaPhase 1
  • Surabgene LomparvovecABBV-RGX-314 / VEGF / Gene therapy / Eye Care
    wet AMD (subretinal)Phase 3wet AMD (suprachoroidal)Phase 2Diabetic Retinopathy (suprachoroidal)Phase 2
  • UbrogepantUBRELVY / CGRP receptor / Small molecule / Neuroscience
    Menstrual MigrainePhase 3
  • ABBV-2001ABBV-2001 / BCMA, CD38, CD3 / Biologic trispecific T-cell engager / Oncology
    Relapsed/Refractory Multiple MyelomaPhase 1
  • Pivekimab SunirinePVEK / CD123 / Biologic ADC / Oncology
  • Mirvetuximab soravtansineELAHERE / FR-alpha / Biologic ADC / Oncology
    Platinum-Resistant Ovarian CancerApprovedPlatinum-Sensitive Ovarian CancerPhase 3
  • AtogepantQULIPTA / CGRP receptor / Small molecule / Neuroscience
    Acute MigraineSubmittedOUSMenstrual MigrainePhase 3OUS
  • EmraclidineM4 PAM / Small molecule / Neuroscience
    SchizophreniaPhase 2PsychosisPhase 1
  • UpadacitinibABT-494 / RINVOQ / JAK1 / Small molecule / Immunology
    Alopecia AreataSubmittedUSEUHidradenitis SuppurativaPhase 3Systemic Lupus ErythematosusPhase 3
  • ABBV-166 (Skyrizi + Lutikizumab)ABBV-166 / SKYRIZI / IL-23 + IL-1a/1b / Biologic combination / Immunology
    Psoriatic ArthritisPhase 2
  • ABBV-848ABBV-848 / IRAK4 / Small molecule / Immunology
    Rheumatoid ArthritisPhase 1
  • ABBV-706ABBV-706 / SEZ6 / Biologic ADC / Oncology
    First Line Small Cell Lung CancerPhase 2Relapsed/Refractory Small Cell Lung CancerPhase 3Neuroendocrine NeoplasmsPhase 1
  • ABBV-1480ABBV-1480 / PD-1, VEGF / Biologic bispecific antibody / Oncology
    Solid TumorsPhase 1
  • LutikizumabABT-981 / IL-1a/1b / Biologic / Immunology
    Hidradenitis SuppurativaPhase 3
  • GemibotulinumtoxinAGemibotA / SNAP-25 / Biologic / Neuroscience
    Ventral Hernia RepairPhase 2
32assets
  • camizestrant + CDK4/6ingSERD + CDK4/6i / small molecule combination / Oncology
    1L HR+ HER2- ESR1m advanced breast cancerPhase IIISubmitted
  • camizestrant + palbociclibngSERD + CDK4/6i / small molecule combination / Oncology
    1L HR+ HER2- advanced breast cancerPhase III
  • camizestrant +/- abemaciclibngSERD + CDK4/6i / small molecule combination / Oncology
    adjuvant HR+ HER2- early breast cancerPhase III
  • camizestrantngSERD / small molecule / Oncology
    adjuvant switch HR+ HER2- early breast cancerPhase III
  • AZD0120AZD0120 / CD19/BCMA CAR-T / cell therapy / Oncology/Rare Disease/Respiratory & Immunology
    relapsed/refractory multiple myelomaPhase IIINew to Phase IIIamyloid light-chain amyloidosisPhase Isystemic lupus erythematosusPhase I
  • puxitatug samrotecanB7-H4 TOP1i ADC / antibody-drug conjugate / Oncology
    2-3L B7-H4+ endometrial cancerPhase III
  • rilvegostomigPD-1/TIGIT bispecific mAb / bispecific antibody / Oncology
    HCC; biliary tract; NSCLC; gastric cancerPhase III
  • saruparibPARP1 inhibitor / small molecule / Oncology
    prostate cancer; breast cancerPhase III
  • sonesitatug vedotinCLDN18.2 MMAE ADC / antibody-drug conjugate / Oncology
    CLDN18.2+ gastric cancerPhase III
  • surovatamigAZD0486 / CD19/CD3 T-cell engager / bispecific antibody / Oncology/Respiratory & Immunology
    1L elderly DLBCL; follicular lymphomaPhase IIIB-cell driven autoimmune diseasePhase I
  • torvutatug samrotecanAZD5335 / anti-FRα TOP1i ADC / antibody-drug conjugate / Oncology
    ovarian cancerPhase III
  • volrustomigPD-1/CTLA-4 bispecific mAb / bispecific antibody / Oncology
    cervical cancer; HNSCC; NSCLC; mesotheliomaPhase IIIcolorectal cancer; NSCLC; other solid tumoursPhase II
  • balcinrenone/dapagliflozinMR antagonist/modulator + SGLT2 inhibitor / small molecule combination / Cardiovascular, Renal and Metabolism
    heart failure with CKDPhase IIICKDPhase II
  • baxdrostataldosterone synthase inhibitor / small molecule / Cardiovascular, Renal and Metabolism
    hypertensionPhase IIIAcceptedprimary aldosteronismPhase III
  • baxdrostat/dapagliflozinaldosterone synthase inhibitor + SGLT2 inhibitor / small molecule combination / Cardiovascular, Renal and Metabolism
    CKD; prevention of heart failurePhase III
  • laroprovstatPCSK9 inhibitor / small molecule / Cardiovascular, Renal and Metabolism
    dyslipidaemiaPhase III
  • WainuaWainua / ligand-conjugated antisense / antisense oligonucleotide / Cardiovascular, Renal and Metabolism
    ATTRv-PNPhase IIILaunchedATTR CMLCM
  • zibotentan/dapagliflozinendothelin A receptor antagonist/SGLT2 inhibitor / small molecule combination / Cardiovascular, Renal and Metabolism
    CKD with high proteinuriaPhase III
  • elecoglipronoral GLP-1 receptor agonist / small molecule / Cardiovascular, Renal and Metabolism
    T2D/chronic weight managementPhase II
  • AZD6234AZD6234 / peptide / peptide / Cardiovascular, Renal and Metabolism
    chronic weight management in overweight or obesityPhase II
  • tozorakimabIL-33 mAb / monoclonal antibody / Respiratory & Immunology
    COPDPhase IIIsevere viral lower respiratory tract diseasePhase IIIasthmaPhase II
  • TezspireTezspire / TSLP mAb / monoclonal antibody / Respiratory & Immunology
    severe uncontrolled asthmaPhase IIILaunchedChinaeosinophilic esophagitis; COPD; nasal polypsLCM
  • SaphneloSaphnelo / type I IFN receptor mAb / monoclonal antibody / Respiratory & Immunology
    systemic lupus erythematosusPhase IIILaunchedChinacutaneous lupus erythematosus; SLE SCLCM
  • anselamimabfibril-reactive mAb / monoclonal antibody / Rare Disease
    amyloid light-chain amyloidosisPhase IIISubmitted
  • cliramitugtransthyretin depleter / large molecule / Rare Disease
    transthyretin amyloid cardiomyopathyPhase III
  • efzimfotase alfanext generation TNSALP ERT / enzyme replacement therapy / Rare Disease
    hypophosphatasiaPhase III
  • eneboparatideparathyroid hormone receptor 1 / large molecule / Rare Disease
    hypoparathyroidismPhase III
  • gefurulimabanti-C5 dual-binding nanobody / nanobody / Rare Disease
    generalised myasthenia gravisPhase IIISubmitted
  • KavigaleKavigale / SARS-CoV-2 LAAB / long-acting antibody / Infectious Disease
    prevention of COVID-19Phase IIILaunched
  • AZD0292AZD0292 / pseudomonas Psl-PcrV bispecific mAb / bispecific antibody / Infectious Disease
    bronchiectasisPhase II
  • AZD5148AZD5148 / anti-Clostridioides difficile TcdB mAb / monoclonal antibody / Infectious Disease
    reduction of C. diff recurrencePhase II
  • AZD7760AZD7760 / S. aureus virulence factors / mAb combination / Infectious Disease
    prevention of Staph aureus infectionPhase II
30assets
  • erenumab-aooeAIMOVIG / CGRP receptor / Monoclonal antibody / Neuroscience
    Pediatric migraineInvestigational
  • blinatumomabBLINCYTO / CD19 x CD3 / BiTE molecule / Hematology/Oncology; Inflammation
    Newly diagnosed B-cell precursor acute lymphoblastic leukemiaInvestigationalRelapsed/refractory acute lymphoblastic leukemia, subcutaneous administrationPhase 2InvestigationalSystemic lupus erythematosus with and without nephritisInvestigational
  • dazodalibepCD40L / Fusion protein / Rare Disease
    Sjögren’s diseaseInvestigational
  • romosozumab-aqqgEVENITY / Sclerostin / Monoclonal antibody / Bone
    Pediatric osteogenesis imperfectaInvestigational
  • tarlatamab-dlleIMDELLTRA / DLL3 x CD3 / BiTE molecule / Hematology/Oncology
    Small cell lung cancer, limited-stage and extensive-stage settingsInvestigationalSmall cell lung cancer, combination studiesInvestigationalSmall cell lung cancer, subcutaneous administrationPhase 1Investigational
  • pegloticaseKRYSTEXXA / Uric acid / PEGylated uricase / Rare Disease
    Uncontrolled gout, subcutaneous administration with methotrexateInvestigational
  • sotorasibLUMAKRAS / KRAS G12C / Small molecule / Hematology/Oncology
    KRAS G12C-mutated metastatic colorectal cancer, first line combinationInvestigationalPreviously treated advanced KRAS G12C-mutated non-small cell lung cancerInvestigationalNewly diagnosed advanced KRAS G12C-mutated NSCLC, combinationInvestigational
  • maridebart cafraglutideAMG 133 / MariTide / GLP-1 receptor; GIP receptor / Antibody-peptide conjugate / Cardiometabolic; Diabetes
    ObesityInvestigationalCardiovascular risk reduction in ASCVD with obesity or overweightInvestigationalHeart failure events and cardiovascular risk reduction in HFpEF/HFmrEF with obesityInvestigational
  • romiplostimNPLATE / Thrombopoietin receptor / Peptibody / Hematology/Oncology
    Chemotherapy-induced thrombocytopeniaInvestigational
  • olpasiranAMG 890 / Lp(a) / siRNA / Cardiometabolic
    Atherosclerotic cardiovascular diseaseInvestigational
  • apremilastOTEZLA / PDE4 / Small molecule / Inflammation
    Juvenile psoriatic arthritisInvestigationalPediatric Behcet’s diseaseInvestigational
  • etelcalcetidePARSABIV / Calcium-sensing receptor / Peptide / Nephrology
    Pediatric secondary hyperparathyroidism in CKD receiving hemodialysisInvestigational
  • evolocumabREPATHA / PCSK9 / Monoclonal antibody / Cardiometabolic
    Hypercholesterolemia in high cardiovascular risk without prior MI or strokeInvestigational
  • avacopanTAVNEOS / C5aR1 / Small molecule / Rare Disease
    Pediatric ANCA-associated vasculitisInvestigational
  • teprotumumab-trbwTEPEZZA / IGF-1R / Monoclonal antibody / Rare Disease
    Thyroid eye disease, subcutaneous administrationInvestigational
  • tezepelumab-ekkoTEZSPIRE / TSLP / Monoclonal antibody / Inflammation
    Chronic obstructive pulmonary diseaseInvestigationalEosinophilic esophagitisInvestigational
  • xaluritamigAMG 509 / STEAP1 x CD3 / XmAb bispecific T-cell engager antibody / Hematology/Oncology
    Prostate cancerInvestigationalEwing sarcomaInvestigational
  • ABP 206ABP 206 / PD-1 / Monoclonal antibody biosimilar / Hematology/Oncology
    Investigational biosimilar to OPDIVO (nivolumab)Investigational biosimilar
  • ABP 234ABP 234 / PD-1 / Monoclonal antibody biosimilar / Hematology/Oncology
    Investigational biosimilar to KEYTRUDA (pembrolizumab)Investigational biosimilar
  • ABP 692ABP 692 / CD20 / Monoclonal antibody biosimilar / Inflammation
    Investigational biosimilar to OCREVUS (ocrelizumab)Investigational biosimilar
  • daxdilimabILT7 / Monoclonal antibody / Rare Disease
    Dermatomyositis and anti-synthetase inflammatory myositisInvestigationalDiscoid lupus erythematosusInvestigational
  • inebilizumabUPLIZNA / CD19 / Monoclonal antibody / Inflammation
    Systemic lupus erythematosus with nephritisInvestigational
  • carfilzomibKYPROLIS / Proteasome / Small molecule / Hematology/Oncology
    Pediatric acute lymphoblastic leukemiaInvestigational
  • AMG 104AMG 104 / TSLP / Monoclonal antibody Fab / Inflammation
    AsthmaInvestigational
  • AMG 732AMG 732 / IGF-1R / Monoclonal antibody / Rare Disease
    Thyroid eye diseaseInvestigational
  • AMG 355AMG 355 / CCR8 / Monoclonal antibody / Hematology/Oncology
    Advanced solid tumor malignanciesInvestigational
  • AMG 410AMG 410 / Small molecule / Hematology/Oncology
    Solid tumorsInvestigational
  • AMG 436AMG 436 / Small molecule / Hematology/Oncology
    Advanced solid tumor malignanciesInvestigational
  • AMG 513AMG 513 / Cardiometabolic
    ObesityInvestigational
  • AMG 691AMG 691 / Monoclonal antibody / Inflammation
    AsthmaInvestigational
30assets
  • Glucose sensitive insulinNN1845 / Analogue / Diabetes
    Type 1 and 2 diabetesPhase 1
  • Ventus NLRP3iNN6022 / NLRP3 / Inhibitor / Diabetes
    CVDPhase 1
  • siRNA GalXC-GYS2siRNA / Diabetes
    siRNA GalXC-GYS2Phase 1
  • CNPCNP / Analogue / Diabetes
    Heart failurePhase 1
  • Amylin 355Analogue / Obesity
    ObesityPhase 1
  • Amylin 1213Analogue / Obesity
    ObesityPhase 1
  • SLC25A5SLC25A5 / siRNA / Obesity
    MASHPhase 1
  • Inno8Antibody fragment / Rare Blood Disorders
    HaemophiliaPhase 1
  • Subcutaneous ZenagamtideGLP-1 / amylin / Co-agonist / Diabetes / Obesity
    Subcutaneous ZenagamtidePhase 2ObesityPhase 2
  • Monlunabant in DKDCB1 receptor / Small molecule / Diabetes
    Diabetic Kidney Disease (DKD)Phase 2
  • Oral ZenagamtideGLP-1 / amylin / Co-agonist / Diabetes / Obesity
    DiabetesPhase 2ObesityPhase 2
  • CDR132LmiR-132 / Antisense oligonucleotide / Diabetes
    Heart failure with reduced ejection fraction (HFrEF)Phase 2
  • ObesityPhase 2
  • TripleGLP1 / GIP / amylin / Tri-agonist / Obesity
    ObesityPhase 2
  • MonlunabantINV-202 / CB1 receptor / Small molecule / Obesity
    ObesityPhase 2
  • NDecEPI01 / Rare Blood Disorders
    Sickle cell disease / An oral combination of decitabine and tetrahydrouridine. Project is developed in collaboration with EpiDestiny.Phase 2
  • EtavopivatRare Blood Disorders
    ThalassemiaPhase 2
  • CagriSema in T2DNN9388 / Analogue / Diabetes
    Type 2 diabetesPhase 3
  • Ziltivekimab ASCVDNN6018 / Diabetes
    Atherosclerotic cardiovascular disease (ASCVD) with chronic kidney disease (CKD) and inflammationPhase 3
  • CoramitugPRX004 / Diabetes
    ATTR CardiomyopathyPhase 3
  • Heart failure with preserved ejection fraction (HFpEF)Phase 3
  • Acute Myocardial infarction (AMI)Phase 3
  • CagrilintideAnalogue / Obesity
    ObesityPhase 3
  • Efruxifermin in MASHFGF21 / Analogue / Obesity
    MASHPhase 3
  • Etavopivat (Sickle Cell Disease)Small molecule / Rare Blood Disorders
    Sickle Cell DiseasePhase 3
  • IcosemaNN1535 / Analogue / Diabetes
    Type 2 diabetes / A combination of GLP-1 analogue semaglutide and insulin icodec intended for once-weekly treatment.Filed
  • Insulin icodecNN1436 / Analogue / Diabetes
    DiabetesFiled
  • CagriSemaNN9898 / Analogue / Obesity
    ObesityFiled
  • Semaglutide 7.2 mgAnalogue / Obesity
    ObesityFiled
  • DenecimigMim8 / Inhibitor / Rare Blood Disorders
    Haemophilia A with or without inhibitors / A next generation FVIII mimetic bispecific antibody for subcutaneous prophylaxis of haemophilia A regardless of inhibitor status.Filed
26assets
  • ArexvyArexvy / RSV F protein / Recombinant protein, adjuvanted vaccine / Infectious diseases
    RSV adults (50-59 years of age at increased risk)Phase III/Registration
  • gepotidacinGSK2140944 / Bacterial topoisomerase inhibitor / Small molecule / Infectious diseases
    Urogenital gonorrhoeaPhase III/Registration
  • bepirovirsenGSK3228836 / HBsAg / Antisense oligonucleotide / Infectious diseases
    Chronic HBV infectionPhase III
  • BexseroBexsero / MenB / Recombinant protein, OMV vaccine / Infectious diseases
    Meningitis B (infants US)Phase III/RegistrationUS
  • MenABCWY vaccineGSK3536819 / Meningococcal ABCWY / Recombinant protein, OMV, conjugated vaccine / Infectious diseases
    Meningococcal diseasePhase III/Registration
  • Nucala (mepolizumab)Nucala / IL-5 / Monoclonal antibody / Respiratory/Immunology
    COPD with eosinophilic phenotypePhase III/Registration
  • depemokimabGSK3511294 / IL-5 / Monoclonal antibody / Respiratory/Immunology
    Asthma with type 2 inflammationPhase III/RegistrationChronic rhinosinusitis with nasal polypsPhase III/RegistrationEGPAPhase III
  • camlipixantGSK4523199 / P2X3 / Small molecule antagonist / Respiratory/Immunology
    Refractory chronic coughPhase III
  • linerixibatGSK2330672 / IBAT / Small molecule / Respiratory/Immunology
    Cholestatic pruritus in primary biliary cholangitisPhase III
  • Blenrep (belantamab mafodotin)GSK2857916 / Blenrep / BCMA / Antibody-drug conjugate / Oncology
    Relapsed/refractory multiple myelomaPhase III/Registration
  • Jemperli (dostarlimab)Jemperli / PD-1 / Monoclonal antibody / Oncology
    Endometrial cancerPhase III/RegistrationRectal cancer (dMMR)Phase IINon-small cell lung cancerPhase III
  • Zejula (niraparib)Zejula / PARP / Small molecule / Oncology
    Ovarian cancerPhase III/Registration
  • belrestotugGSK4428859 / TIGIT / Monoclonal antibody / Oncology
    NSCLC and other solid tumorsPhase III
  • GSK5764227GSK5764227 / B7-H3 / Antibody-drug conjugate / Oncology
    Extensive-stage small-cell lung cancerPhase IIIAdvanced GI solid tumorsPhase I/II
  • cobolimabGSK4069889 / TIM-3 / Monoclonal antibody / Oncology
    Non-small cell lung cancerPhase II/III
  • GSK4532990GSK4532990 / HSD17B13 / RNA interference / Opportunity driven
    NASH/MASHPhase II
  • GSK4172239GSK4172239 / DNMT1 / Small molecule inhibitor / Opportunity driven
    Sickle cell diseasePhase I
  • GSK3882347GSK3882347 / FimH / Small molecule antagonist / Infectious diseases
    Uncomplicated UTIPhase I
  • GSK3923868GSK3923868 / PI4K beta / Small molecule inhibitor / Infectious diseases
    Rhinovirus diseasePhase I
  • GSK3965193GSK3965193 / PAPD5/PAPD7 / Small molecule inhibitor / Infectious diseases
    Chronic HBV infectionPhase I
  • GSK2556286GSK2556286 / Mtb cholesterol-dependent / Small molecule inhibitor / Infectious diseases
    TuberculosisPhase I
  • GSK3772701GSK3772701 / P. falciparum whole cell / Small molecule inhibitor / Infectious diseases
    MalariaPhase I
  • GSK4024484GSK4024484 / P. falciparum whole cell / Small molecule inhibitor / Infectious diseases
    MalariaPhase I
  • GSK3536867GSK3536867 / Salmonella typhoid + paratyphoid A / Bivalent conjugate vaccine / Infectious diseases
    Salmonella (typhoid + paratyphoid A)Phase I
  • AGN-151586 / progranulin program (latozinemab)AL001 / Progranulin/sortilin / Monoclonal antibody / Opportunity driven
    Frontotemporal dementia with progranulin gene mutationPhase III
26assets
  • ZanubrutinibBTK / Inhibitor / Hematologic Malignancies / Immunology & Inflammation
    Treatment-Naive Chronic Lymphocytic Leukemia / Small Lymphocytic LymphomaApprovedRelapsed/Refractory Chronic Lymphocytic Leukemia / Small Lymphocytic LymphomaApprovedRelapsed/Refractory Waldenström Macroglobulinemia and Treatment-Naïve Waldenström MacroglobulinemiaApproved
  • SonrotoclaxBCL2 / Inhibitor / Hematologic Malignancies
    Treatment-Naive Chronic Lymphocytic LeukemiaPhase 3Treatment-Naive Chronic Lymphocytic LeukemiaPhase 3Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic LymphomaApproved
  • TislelizumabPD-1 / Monoclonal Antibody / Lung Cancers / Gastrointestinal Cancers / Other Cancers
    First-Line Extensive-Stage Small Cell Lung CancerApprovedFirst-Line Non-Squamous Non-Small Cell Lung CancerApprovedFirst-Line Squamous Non-Small Cell Lung CancerApproved
  • BGB-16673BTK Chimeric Degradation Activation Compound [CDAC] / Hematologic Malignancies / Immunology & Inflammation
    Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic LymphomaPhase 3Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic LymphomaPhase 3Relapsed/Refractory Mantle Cell LymphomaPhase 2
  • BlinatumomabCD19 x CD3 / BiTE® [Bispecific T-Cell Engager] / Hematologic Malignancies
    Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (subcutaneous formulation)Phase 2
  • TarlatamabDLL3 x CD3 / BiTE® [Bispecific T-cell Engager] / Lung Cancers
    First-Line Extensive-Stage Small Cell Lung CancerPhase 3First-Line Extensive-Stage Small Cell Lung CancerPhase 3Second-Line Small Cell Lung CancerPhase 3
  • ZanidatamabHER2 Bispecific / Antibody / Gastrointestinal Cancers
    First-Line HER2 Positive Gastroesophageal AdenocarcinomaPhase 3Second-Line HER2 Positive Biliary Tract CancerApprovedFirst-Line HER2 positive Biliary Tract CancerPhase 3
  • BG-68501CDK2 / Inhibitor / Breast/Gynecologic Cancers
    Breast Cancer and Solid TumorsPhase 1
  • BGB-21447BCL2 / Inhibitor / Breast/Gynecologic Cancers
    Hormone Receptor Positive Breast CancerPhase 1
  • BGB-43395CDK4 / Inhibitor / Breast/Gynecologic Cancers
    Breast Cancer and Solid TumorsPhase 1
  • BGB-B455Claudin 6 x CD3 Bispecific / Antibody / Breast/Gynecologic Cancers
    Gynecologic and Solid TumorsPhase 1
  • BG-C9074B7-H4 Antibody Drug Conjugate / Breast/Gynecologic Cancers
    Breast Cancer and Solid TumorsPhase 1
  • BG-75202KAT6A/B / Inhibitor / Breast/Gynecologic Cancers
    Breast CancerPhase 1
  • BG-75098CDK2 Chimeric Degradation Activation Compound [CDAC] / Breast/Gynecologic Cancers
    Breast CancerPhase 1
  • BGB-B2033GPC3 x 4-1BB Bispecific / Antibody / Gastrointestinal Cancers
    Solid TumorsPhase 1
  • BG-C137FGFR2b Antibody Drug Conjugate / Gastrointestinal Cancers
    Gastrointestinal CancersPhase 1
  • BGB-53038Pan-KRAS / Inhibitor / Gastrointestinal Cancers
    Lung Cancers and GI CancersPhase 1
  • BG-C477CEA Antibody Drug Conjugate / Gastrointestinal Cancers
    Lung Cancers and GI CancersPhase 1
  • BG-C0902EGFR x MET x MET Antibody Drug Conjugate / Lung Cancers
    Lung Cancers and GI CancersPhase 1
  • BG-T187EGFR x MET x MET Trispecific / Antibody / Lung Cancers
    Lung Cancers and GI CancersPhase 1
  • BGB-58067MTA Cooperative PRMT5 / Inhibitor / Lung Cancers
    Lung Cancers and GI CancersPhase 1
  • BG-89894MAT2A / Inhibitor / Lung Cancers
    Lung Cancers and Solid TumorsPhase 1
  • BG-60366EGFR Chimeric Degradation Activation Compound [CDAC] / Lung Cancers
    Lung CancersPhase 1
  • BGB-26808HPK1 / Inhibitor / Gastrointestinal Cancers
    Solid Tumors/Pan- TumorPhase 1
  • XaluritamigSTEAP1 x CD3 Bispecific T-cell Engager (XmAb®) / Other Cancers
    Metastatic Castration-Resistant Prostate CancerPhase 1
  • BGB-45035IRAK4 Chimeric Degradation Activation Compound [CDAC] / Immunology & Inflammation
    Rheumatoid ArthritisPhase 2Skin DiseasesPhase 1
24assets
  • NucresiranRNAi therapeutic / Transthyretin Amyloidosis (TTR)
    ATTR Amyloidosis with CardiomyopathyPhase 3hATTR Amyloidosis with PolyneuropathyPhase 3
  • ZilebesiranRNAi therapeutic / Cardiovascular
    HypertensionPhase 3REVERSIR HypertensionPhase 1
  • RapirosiranALN-HSD / HSD / RNAi therapeutic / Metabolic
    Metabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 2
  • ALN-ANG3ALN-ANG3 / ANG3 / RNAi therapeutic / Metabolic
    Diabetic Kidney DiseasePhase 2
  • ALN-4324ALN-4324 / GRB14 / RNAi therapeutic / Metabolic
    Type 2 Diabetes MellitusPhase 1
  • ALN-2232ALN-2232 / ACVR1C / RNAi therapeutic / Metabolic
    Obesity & Weight ManagementPhase 1
  • ALN-PNPALN-PNP / PNP / RNAi therapeutic / Metabolic
    Non-Alcoholic Fatty Liver Disease (NAFLD)Phase 1
  • ALN-APOC3ALN-APOC3 / APOC3 / RNAi therapeutic / Metabolic
    DyslipidemiaPhase 1
  • ALN-CIDEBALN-CIDEB / CIDEB / RNAi therapeutic / Metabolic
    Metabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 1
  • Cemdisiran(+/- Pozelimab)RNAi therapeutic / Neuroscience/Other
    Myasthenia GravisPhase 3Geographic AtrophyPhase 3
  • MivelsiranALN-APP / APP / RNAi therapeutic / Neuroscience
    Cerebral Amyloid Angiopathy (CAA)Phase 2Alzheimer’s DiseasePhase 1
  • ALN-HTT02ALN-HTT02 / HTT / RNAi therapeutic / Neuroscience
    Huntington’s DiseasePhase 1
  • ALN-5288ALN-5288 / MAPT / RNAi therapeutic / Neuroscience
    Alzheimer’s DiseasePhase 1
  • ALN-SODALN-SOD / SOD1 / RNAi therapeutic / Neuroscience
    SOD1 Amyotrophic Lateral Sclerosis (ALS)Phase 1
  • ALN-SNCAALN-SNCA / SNCA / RNAi therapeutic / Neuroscience
    Parkinson’s DiseasePhase 1
  • Cemdisiran (+Pozelimab)RNAi therapeutic / Hematology
    Paroxysmal Nocturnal HemoglobinuriaPhase 3
  • ALN-6400ALN-6400 / PLG / RNAi therapeutic / Hematology
    Bleeding DisordersPhase 2
  • AG-236ALN-TMP / TMP / RNAi therapeutic / Hematology
    Polycythemia VeraPhase 1
  • ALN-CFBALN-CFB / CFB / RNAi therapeutic / Hematology
    Paroxysmal Nocturnal HemoglobinuriaPhase 1
  • Elebsiran (+Tobevibart)RNAi therapeutic / Other
    Hepatitis D Virus InfectionPhase 2
  • ALN-BCATALN-BCAT / BCAT / RNAi therapeutic / Other
    Hepatocellular CarcinomaPhase 1
  • ALN-4285ALN-4285 / RNAi therapeutic / Other
    Healthy VolunteersPhase 1
  • ALN-4915ALN-4915 / RNAi therapeutic / Other
    Healthy VolunteersPhase 1
  • ALN-F1202ALN-F1202 / RNAi therapeutic / Other
    Healthy VolunteersPhase 1
21assets
  • PumitamigPD-L1/VEGF-A / immunomodulator / Oncology
    Multiple solid tumors; NSCLC; SCLC; HCC; CRC; ES-SCLC; EGFRm NSCLC; glioblastoma; MPM; NEN; TNBC; PDACclinical
  • BNT325/DB-1305BNT325/DB-1305 / targeted therapy / Oncology
    Multiple solid tumorsclinical
  • BNT324/DB-1311BNT324/DB-1311 / targeted therapy / Oncology
    Multiple solid tumorsclinical
  • BNT321BNT321 / targeted therapy / Oncology
    Multiple solid tumors; HCC combinationclinical
  • Autogene cevumeranmRNA cancer immunotherapy / Oncology
    Adjuvant CRC; adjuvant PDACclinical
  • BNT116BNT116 / mRNA cancer immunotherapy / Oncology
    Advanced NSCLC; 1L advanced NSCLCclinical
  • BNT113BNT113 / HPV16 / mRNA cancer immunotherapy / Oncology
    1L HPV16+ HNSCCclinical
  • GotistobartCTLA-4 / immunomodulator / Oncology
    Metastatic NSCLC; metastatic CRPC; PROC; multiple solid tumorsclinical
  • BNT211BNT211 / cell therapy / Oncology
    Multiple solid tumorsclinical
  • BNT314/GEN1059BNT314/GEN1059 / immunomodulator / Oncology
    Multiple solid tumors; metastatic CRC combinationclinical
  • BNT329BNT329 / immunomodulator / Oncology
    Multiple solid tumorsclinical
  • BNT326/YL202BNT326/YL202 / targeted therapy / Oncology
    Multiple solid tumors; NSCLC combination; advanced/metastatic breast cancerclinical
  • BNT317BNT317 / immunomodulator / Oncology
    Multiple solid tumorsclinical
  • Trastuzumab pamirtecanHER2 / targeted therapy / Oncology
    Metastatic breast cancer; advanced/metastatic breast cancer combination; 2L endometrial cancer; multiple solid tumorsclinical
  • BNT162 + BNT161BNT162 + BNT161 / mRNA vaccine combination / Infectious diseases
    COVID-19-influenza combinationclinical
  • BNT163BNT163 / HSV / mRNA vaccine / Infectious diseases
    HSVclinical
  • BNT166BNT166 / Mpox / mRNA vaccine / Infectious diseases
    Mpoxclinical
  • BNT162BNT162 / COMIRNATY / SARS-CoV-2 / mRNA vaccine / Infectious diseases
    COVID-19Commercialmarketed
  • BNT351BNT351 / HIV / antibody mRNA / Infectious diseases
    HIVclinical
  • BNT164BNT164 / Tuberculosis / mRNA vaccine / Infectious diseases
    Tuberculosisclinical
  • BNT165BNT165 / Malaria / mRNA vaccine / Infectious diseases
    Malariaclinical
19assets
  • LenacapavirHIV capsid inhibitor / Long-acting injectable / Virology
    HIV Prevention (PrEP, PURPOSE 365)Phase 3HIV Treatment (in combinations)Phase 3HIV Treatment (with bNAbs)Phase 2
  • Bictegravir/lenacapavir oral combinationINSTI + capsid inhibitor / Oral / Virology
    HIV Treatment (virologically suppressed, ARTISTRY-1 & ARTISTRY-2)Phase 3
  • Islatravir/lenacapavir oral combinationLong-acting oral / Virology
    HIV Treatment (virologically suppressed, ISLEND-1 & ISLEND-2)Phase 3
  • Lenacapavir + teropavimab + zinlirvimabCapsid + bNAbs / Long-acting injectable / Virology
    HIV Treatment (virologically suppressed)Phase 2
  • GS-1720/GS-4182GS-1720/GS-4182 / INSTI/capsid inhibitor / Long-acting oral / Virology
    HIV Treatment (WONDERS-1 & -2)Phase 2
  • Teropavimab + zinlirvimabbNAbs / Broadly neutralizing antibodies / Virology
    HIV CurePhase 2
  • VesatolimodVirology
    HIV Cure (FRESH)Phase 2
  • HBV CurePhase 2
  • HepcludexHepcludex / Virology
    Chronic HDV with compensated liver disease (MYR301)FiledBLA pending (US); Approved (EU)USEU
  • EdecesertibInflammation
    Lupus (COSMIC)Phase 2
  • Inflammatory bowel disease (PALEKONA)Phase 2
  • α4β7 inhibitorα4β7 / Inflammation
    Inflammatory Bowel Disease (SWIFT)Phase 2
  • Sacituzumab govitecan-hziyAntibody-drug conjugate / Oncology
    1L metastatic triple-negative breast cancer (PD-L1-, ASCENT-03)FiledsBLA pendingUS1L metastatic triple-negative breast cancer (PD-L1+, with pembrolizumab, ASCENT-04)FiledsBLA pendingUSHigh risk adjuvant triple-negative breast cancer (with pembrolizumab, ASCENT-05)Phase 3
  • Axicabtagene ciloleucelCD19 CAR / Cell therapy / Oncology
    2L+ high-risk follicular lymphoma (ZUMA-22)Phase 31L high-risk large B-cell lymphoma (ZUMA-23)Phase 3
  • Brexucabtagene autoleucelCD19 CAR / Cell therapy / Oncology
    Pediatric acute lymphocytic leukemia/non-Hodgkin lymphoma (ZUMA-4)Phase 2
  • Anitocabtagene autoleucelCell therapy / Oncology
    2-4L relapsed/refractory multiple myeloma (iMMagine-3)Phase 34L+ relapsed/refractory multiple myeloma (iMMagine-1)FiledBLA pendingUS
  • KITE-363KITE-363 / CD19/CD20 bicistronic / Cell therapy / Oncology
    Relapsed/refractory diffuse large B-cell lymphomaPhase 1RMAT designationRheumatology (autoimmune)Phase 1
  • DenikitugGS-1811 / Oncology
    Advanced cancersPhase 1
  • Domvanalimab + zimberelimabAnti-TIGIT + anti-PD1 / Combination therapy / Oncology
    1L metastatic non-small cell lung cancer (with chemo, STAR-121)Phase 31L head and neck squamous cell carcinoma (VELOCITY-HNSCC)Phase 2
19assets
  • ZilganersenION373 / GFAP / RNA-Targeted Therapeutic / Neurology
    Alexander DiseasePhase 1/3ActiveGlobal
  • ION582ION582 / UBE3A-ATS / RNA-Targeted Therapeutic / Neurology
    Angelman SyndromeClinicalActive
  • ION269ION269 / APP / RNA-Targeted Therapeutic / Neurology
    Alzheimer's DiseaseClinicalActive
  • ION717ION717 / PRNP / RNA-Targeted Therapeutic / Neurology
    Prion DiseaseClinicalActive
  • ION356ION356 / PLP1 / RNA-Targeted Therapeutic / Neurology
    Pelizaeus-Merzbacher DiseaseClinicalActive
  • ION859ION859 / LRRK2 / RNA-Targeted Therapeutic / Neurology
    Parkinson's DiseaseClinicalActive
  • ION464ION464 / SNCA / RNA-Targeted Therapeutic / Neurology
    Multiple System AtrophyClinicalActiveParkinson's DiseaseClinicalActive
  • ION440ION440 / MECP2 / RNA-Targeted Therapeutic / Neurology
    MECP2 Duplication SyndromeClinicalActive
  • OlezarsenApoC-III / RNA-Targeted Therapeutic / Cardiology
    Severe HypertriglyceridemiaClinicalActive
  • DonidalorsenPKK / RNA-Targeted Therapeutic / Specialty/Rare
    Hereditary AngioedemaClinicalActive
  • SapablursenTMPRSS6 / RNA-Targeted Therapeutic / Specialty/Rare
    Polycythemia VeraClinicalActive
  • UlefnersenFUS / RNA-Targeted Therapeutic / Neurology
    Amyotrophic Lateral SclerosisClinicalActive
  • TofersenSOD1 / RNA-Targeted Therapeutic / Neurology
    Amyotrophic Lateral Sclerosis (Presymptomatic SOD1)ClinicalActive
  • IONIS-MAPTRxBIIB080 / TAU / RNA-Targeted Therapeutic / Neurology
    Alzheimer's DiseaseClinicalActive
  • TominersenIONIS-HTTRx / HTT / RNA-Targeted Therapeutic / Neurology
    Huntington's DiseasePhase 2Active
  • ION306ION306 / SMN2 / RNA-Targeted Therapeutic / Neurology
    Spinal Muscular AtrophyClinicalActive
  • EplontersenTTR / RNA-Targeted Therapeutic / Cardiology
    Transthyretin-Mediated Amyloid CardiomyopathyPhase 3Active
  • ION904ION904 / Angiotensinogen / RNA-Targeted Therapeutic / Cardiology
    Treatment-Resistant HypertensionClinicalActive
  • BepirovirsenHepatitis B Virus / RNA-Targeted Therapeutic / Other Medicines
    Hepatitis B Virus InfectionPhase 3Active
18assets
  • zolbetuximabIMAB362 / VYLOY / Claudin 18.2 / Antibody / Oncology
    Gastric and gastroesophageal junction adenocarcinoma, combo with pembrolizumab and chemotherapyFiledEuropeJapan
  • gilteritinibASP2215 / XOSPATA / FLT3 / Small molecule / Oncology
    Newly diagnosed acute myeloid leukemia with high intensity induction chemotherapyPhase 3Post-chemotherapy maintenance acute myeloid leukemiaPhase 3Post-hematopoietic stem cell transplant maintenance acute myeloid leukemiaPhase 3
  • enfortumab vedotinASG-22ME / PADCEV / Nectin-4 / Antibody-drug conjugate (ADC) / Oncology
    Cisplatin-eligible muscle-invasive bladder cancer, combo with pembrolizumabFiledEuropeUSCisplatin-ineligible muscle-invasive bladder cancer, combo with pembrolizumabFiledEuropeJapanBladder-sparing muscle-invasive bladder cancer, combo with pembrolizumabPhase 2
  • fezolinetantESN364 / VEOZAH / NK3 receptor / Small molecule / Women's Health
    Vasomotor symptoms due to menopausePhase 3ChinaVasomotor symptoms in breast cancer patients on adjuvant endocrine therapyPhase 2
  • zocaglusagene nuzaparvovecAT845 / GAA gene replacement / Gene therapy (AAV-based gene therapy) / Genetic Regulation
    Pompe diseasePhase 1
  • ASP2957ASP2957 / MTM1 gene replacement / Gene therapy (AAV-based gene therapy) / Genetic Regulation
    X-linked myotubular myopathyPhase 1
  • setidegrasibKRAS G12D / Small molecule degrader / Targeted Protein Degradation
    Pancreatic ductal adenocarcinomaPhase 3
  • ASP7317ASP7317 / Retinal pigment epithelial cells / Cell therapy / Ophthalmology
    Geographic atrophy secondary to age-related macular degenerationPhase 1
  • ASP1002ASP1002 / Claudin 4/CD137 / T-cell engager bispecific antibody / Oncology
    Gastric and gastroesophageal junction adenocarcinoma; pancreatic adenocarcinomaPhase 1
  • ASP5834ASP5834 / Pan-KRAS / Small molecule degrader / Oncology
    CancerPhase 1
  • VIR-5500VIR-5500 / PSMA/CD3 / T-cell engager / Oncology
    Prostate cancerPhase 1
  • ASP2998ASP2998 / TROP2 / Antibody-drug conjugate (ADC) / Oncology
    CancerPhase 1
  • ASP2138ASP2138 / Claudin 18.2/CD3 / T-cell engager bispecific antibody / Oncology
    CancerPhase 2
  • ASP546CASP546C / Claudin 18.2 / Antibody-drug conjugate (ADC) / Oncology
    CancerPhase 2Europe
  • abiraterone decanoateASP5541/PRL-02 / CYP17 lyase / Small molecule / Oncology
    Prostate cancerPhase 2
  • mirabegronYM178 / Beta-3 receptor / Small molecule / Urology
    Neurogenic detrusor overactivity in pediatric patients aged 6 months to less than 3 yearsPhase 3Europe
  • roxadustatASP1517/FG-4592 / HIF-PH / Small molecule / Nephrology
    Anemia associated with chronic kidney disease in pediatric patientsPhase 3Europe
  • pudexacianinium chlorideASP5354 / Drug-device combination / Other
    Intraoperative ureter visualization for minimally invasive and open abdominopelvic surgeriesPhase 3
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  • Mesalamine DRAsacol HD / generic small molecule / Gastroenterology
  • EverolimusAfinitor / mTOR / generic small molecule / Oncology
  • Prednisolone acetatePred-Forte / generic small molecule / Ophthalmology
  • EltrombopagPromacta / TPO receptor agonist / generic small molecule / Hematology
  • EpinephrineAdrenalin / adrenergic receptors / generic small molecule / Emergency/Critical Care
  • LenalidomideRevlimid / generic small molecule / Oncology
  • Risperidone ERRisperdal Consta / dopamine/serotonin receptors / generic long-acting injectable / Psychiatry
  • Romidepsin injectionRomidepsin / HDAC / generic small molecule / Oncology
  • CyclosporineRestasis / calcineurin / generic small molecule / Ophthalmology
  • Beclomethasone dipropionateQVAR / glucocorticoid receptor / generic inhaled corticosteroid / Respiratory
  • Sodium BicarbonateNeut / generic injectable / Critical Care
  • Lanreotide injectionSomatuline Depot / somatostatin receptors / generic peptide injectable / Endocrinology/Oncology
  • RifaximinXifaxan / generic small molecule / Gastroenterology
  • Omalizumab biosimilarXOLAIR / IgE / biosimilar monoclonal antibody / Immunology/Allergy
  • Sodium oxybateXyrem / generic small molecule / Neurology
  • BimatoprostLumigan / prostaglandin receptor / generic small molecule / Ophthalmology
  • Denosumab biosimilarsPROLIA & XGEVA / RANKL / biosimilar monoclonal antibody / Osteoporosis/Bone Cancer
  • Albuterol sulfateProAir HFA / beta-2 adrenergic receptor / generic inhaled small molecule / Respiratory
18assets
  • PlozasiranARO-APOC3 / REDEMPLO / APOC3 / RNAi therapeutic / Cardiometabolic
    Familial chylomicronemia syndromeIn MarketSevere hypertriglyceridemiaPhase 3ActiveHypertriglyceridemiaPhase 3Active
  • ZodasiranARO-ANG3 / ANGPTL3 / RNAi therapeutic / Cardiometabolic
    Homozygous familial hypercholesterolemiaPhase 3Recruiting
  • OlpasiranAMG 890; ARO-LPA / LPA / RNAi therapeutic / Cardiovascular disease
    Elevated lipoprotein(a)/cardiovascular riskPhase 3Recruiting/Active
  • GSK4532990ARO-HSD / HSD17B13 / RNAi therapeutic / Liver disease
    MASH/NASHPhase 2Recruiting
  • ARO-PNPLA3JNJ-75220795 / PNPLA3 / RNAi therapeutic / Liver disease
    MASH/NASHPhase 1
  • ARO-INHBEINHBE/Activin E / RNAi therapeutic / Obesity/metabolic disease
    Obesity with or without type 2 diabetesPhase 1
  • ARO-ALK7ACVR1C/ALK7 / RNAi therapeutic / Obesity/metabolic disease
    Obesity with or without type 2 diabetesPhase 1Recruiting
  • ARO-MAPTMAPT/tau / RNAi therapeutic / CNS
    Alzheimer's diseasePhase 1Recruiting
  • ARO-ATXN2ATXN2 / RNAi therapeutic / CNS
    Spinocerebellar ataxia 2Phase 1
  • SRP-1005ARO-HTT / HTT / siRNA therapeutic / CNS
    Huntington's diseasePhase 1
  • ARO-RAGERAGE / RNAi therapeutic / Pulmonary
    Inflammatory pulmonary diseasesPhase 2Active
  • ARO-MMP7MMP7 / RNAi therapeutic / Pulmonary
    Idiopathic pulmonary fibrosisPhase 1
  • FazirsiranTAK-999; ARO-AAT / Z-AAT / RNAi therapeutic / Liver disease
    Alpha-1 antitrypsin deficiency-associated liver diseasePhase 3Recruiting
  • ARO-HBVJNJ-3989; JNJ-73763989 / HBV gene products / RNAi therapeutic / Infectious disease
    Chronic hepatitis BPhase 2
  • ARO-DUX4DUX4 / RNAi therapeutic / Muscular disease
    Facioscapulohumeral muscular dystrophyPhase 2
  • ARO-DM1DMPK mRNA / RNAi therapeutic / Muscular disease
    Myotonic dystrophy type 1Phase 2Recruiting
  • ARO-C3Complement component 3 / RNAi therapeutic / Complement mediated disease
    Complement-mediated renal diseasePhase 1RecruitingC3 glomerulopathyPhase 1RecruitingIgA nephropathyPhase 1Recruiting
  • ARO-CFBComplement factor B / RNAi therapeutic / Complement mediated disease
    Complement mediated diseasePhase 1
18assets
  • Vabysmofaricimab-svoa / VABYSMO / VEGF/Ang-2 / Bispecific antibody / Ophthalmology
    Neovascular age-related macular degeneration (nAMD) and Diabetic macular edema (DME)Approvedapproved
  • Ojemdatovorafenib / OJEMDA / Oncology
    Oncology (undisclosed approved indication)Approvedapproved
  • Miplyffaarimoclomol / MIPLYFFA / Rare Diseases / Neurology
    Niemann-Pick disease type C (NPC)ApprovedapprovedUS
  • Xaciatoclindamycin phosphate vaginal gel 2% / XACIATO / Small molecule / Gynecology / Infectious Disease
    Bacterial VaginosisApprovedapproved
  • Ixinitycoagulation factor IX (recombinant) / IXINITY / Factor IX / Recombinant protein / Hematology
    Hemophilia BApprovedapproved
  • Dsuviasufentanil sublingual tablet / DSUVIA / Opioid receptor / Small molecule / Pain Management
    PainApprovedapproved
  • Dare to Playsildenafil cream / DARE to PLAY / PDE5 / Small molecule / Gynecology / Sexual Health
    Female Sexual Arousal DisorderApprovedapproved
  • ersodetugRZ358 / INSR / Antibody / Metabolic Disorders
    Hypoglycemia due to congenital hyperinsulinism (cHI)Phase 3ongoingHypoglycemia due to tumor hyperinsulinismPhase 3ongoing
  • RZ402RZ402 / Ophthalmology
    Diabetic macular edema (DME)Phase 2ongoing
  • D-FiFCX-007 / Gene Therapy / Dermatology / Rare Diseases
    Dystrophic epidermolysis bullosa (DEB)Phase 3ongoing
  • seralutinibGB002 / PDGFR, CSF1R, and c-KIT / Inhaled tyrosine kinase inhibitor / Respiratory / Cardiovascular
    Pulmonary arterial hypertension (PAH)Phase 3ongoingPulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD)Phase 2ongoing
  • cetrelimabJNJ-63723283 / PD-1 / Antibody / Oncology / Infectious Disease
    Muscle-invasive bladder cancer (MIBC)Phase 2ongoingChronic Hepatitis B Virus InfectionPhase 2ongoing
  • volixibatLUM-002 / Ileal bile acid transporter (IBAT) / Small molecule / Gastroenterology
    Primary sclerosing cholangitis (PSC)Phase 2bongoingPrimary biliary cholangitis (PBC)Phase 2ongoing
  • osavampatorNeurology / Psychiatry
    Major depressive disorderPhase 3ongoing
  • OHB-607OHB-607 / Recombinant human IGF-I/IGFBP-3 / Recombinant protein / Respiratory / Neonatology
    Prevention of bronchopulmonary dysplasia in extremely premature infantsPhase 2ongoing
  • REC-4881REC-4881 / Allosteric MEK1/2 / Small molecule / Oncology
    Familial adenomatous polyposisPhase 2ongoing
  • NIS793NIS793 / Antibody / Oncology
    Solid tumorsPhase 3ongoing
  • QTORIN rapamycinQTORIN / mTOR / Small molecule / Dermatology / Rare Diseases
    Cutaneous venous malformationsPhase 3ongoingClinically significant angiokeratomasPhase 2ongoing
17assets
  • direclidineM4 / Small Molecule / Psychiatry
    SchizophreniaPhase 3Bipolar ManiaPhase 2
  • NBI-570M1/M4 / Small Molecule / Psychiatry
    SchizophreniaPhase 2
  • osavampatorAMPA / Small Molecule / Psychiatry
    Major Depressive DisorderPhase 3
  • NBI-986M4 / Small Molecule / Neurology
    Movement DisordersPhase 1
  • NBI-890VMAT2 / Small Molecule / Psychiatry
    Tardive DyskinesiaPhase 2
  • NBI-569M1/M4 / Small Molecule / Psychiatry
    Alzheimer's PsychosisPhase 1
  • NBI-567M1 / Small Molecule / Neurology
    Alzheimer's CognitionPhase 1
  • NBI-355Nav1.2/1.6 / Small Molecule / Neurology
    EpilepsyPhase 1
  • NBIP-1435CRF1 / Peptide / Endocrinology
    Classic Congenital Adrenal HyperplasiaPhase 1
  • NBIP-2118CRF2 / Peptide / Endocrinology
    ObesityPhase 1
  • NBIP-1968TG + NBIP-2118 Combo / Peptide / Endocrinology
    ObesityPhase 4
  • NBIB-223Frataxin / Gene Therapy / Neurology
    Friedreich's AtaxiaPhase 4
  • NBI-675VMAT2 / Small Molecule / Psychiatry
    CNS IndicationsPhase 1
  • NBI-188CRF1 / Small Molecule / Endocrinology
    Women's HealthPhase 4
  • NBIM-1748Immunology
    UndisclosedPhase 4
  • NBIM-1008Immunology
    UndisclosedPhase 4
  • NBIM-1112Immunology
    UndisclosedPhase 1
17assets
  • FianlimabLAG3 / Monoclonal Antibody / Oncology
    1L metastatic melanoma, adjuvant melanomaPhase 3ActiveGlobal
  • CemiplimabLibtayo / PD-1 / Monoclonal Antibody / Oncology
    CancerPhase 2/3ActiveGlobal
  • DavutamigREGN5093 / METxMET / Bispecific Antibody / Oncology
    CancerPhase 2ActiveGlobal
  • ItepekimabIL-33 / Monoclonal Antibody / Immunology & Inflammation / Respiratory
    COPD, CRSwNPPhase 3ActiveGlobal
  • LinvoseltamabLynozyfic / BCMAxCD3 / Bispecific Antibody / Oncology
    Multiple myelomaPhase 2/3ActiveGlobal
  • MarlotamigREGN7075 / EGFRxCD28 / Bispecific Antibody / Oncology
    Solid TumorsPhase 2ActiveGlobal
  • OdronextamabOrdspono / CD20xCD3 / Bispecific Antibody / Oncology
    LymphomaPhase 2ActiveGlobal
  • UbamatamabREGN4018 / MUC16xCD3 / Bispecific Antibody / Oncology
    Ovarian CancerPhase 2ActiveGlobal
  • NezastomigREGN5678 / PSMAxCD28 / Bispecific Antibody / Oncology
    Prostate CancerPhase 2ActiveGlobal
  • RapirosiranALN-HSD / HSD17B13 / siRNA / Cardiovascular & Metabolic
    NASHPhase 2ActiveGlobal
  • ALN-ANG3ALN-ANG3 / ANGPTL3 / siRNA / Cardiovascular & Metabolic
    DyslipidemiaPhase 2ActiveGlobal
  • CemdisiranC5 / siRNA / Ophthalmology / Immunology
    Geographic atrophyClinicalActiveGlobal
  • PozelimabC5 / Monoclonal Antibody / Ophthalmology / Immunology
    Geographic atrophyClinicalActiveGlobal
  • BetV1REGN5713-5715 / BetV1 / Monoclonal Antibody / Immunology & Inflammation
    Birch AllergyClinicalActiveGlobal
  • DB-OTOOtarmeni / Otoferlin / Gene Therapy / Neurology / Hearing Loss
    Otoferlin-related hearing lossPhase 1/2ActiveGlobal
  • MibavademabUnknown / Biologic / Oncology
    CancerPhase 2ActiveGlobal
  • TrevogrumabREGN1033 / Myostatin / Monoclonal Antibody / Metabolic
    Muscle weakness/wastingPhase 2ActiveGlobal
16assets
  • mezigdomidecereblon E3 ligase / CELMoD; oral targeted protein degrader / Hematology
    relapsed/refractory multiple myelomaPhase IIIactive
  • iberdomidecereblon E3 ligase / CELMoD; oral targeted protein degrader / Hematology
    multiple myelomaPhase IIIactive
  • golcadomidecereblon E3 ligase / CELMoD; oral targeted protein degrader / Hematology
    aggressive B-cell lymphomaPhase IIactive
  • pumitamigBNT327/BMS-986545 / PD-L1; VEGF-A / bispecific antibody / Oncology
    solid tumorsPhase IIIactive
  • iza-brenantibody-drug conjugate / Oncology
    solid tumorsPhase I/IIactive
  • nivolumabOPDIVO / PD-1 / monoclonal antibody / Oncology
    multiple oncology indicationsPhase IIIactive LCM
  • nivolumab and relatlimab HDPD-1; LAG-3 / antibody combination / Oncology
    oncology indicationsPhase IIIactive LCM
  • ipilimumabYERVOY / CTLA-4 / monoclonal antibody / Oncology
    multiple oncology indicationsPhase IIIactive LCM
  • KarXTCOBENFY / muscarinic receptors / small molecule / Neuroscience
    neuropsychiatric indicationsPhase IIIactive LCM
  • milvexianFactor XIa / small molecule anticoagulant / Cardiovascular
    thrombotic disordersPhase IIIactive
  • BMS-986278BMS-986278 / LPA1 / small molecule antagonist / Fibrosis
    idiopathic pulmonary fibrosisPhase IIIactive
  • BMS-986495BMS-986495 / MTBR tau / antibody / Neuroscience
    Alzheimer's diseasePhase IIactive
  • imzokitugCCR8 / monoclonal antibody / Oncology
    solid tumorsPhase I/IIactive
  • obexelimabCD19; FcγRIIb / bifunctional antibody / Immunology
    autoimmune diseasesPhase IIIactive
  • luspatercept-aamtREBLOZYL / TGF-beta superfamily ligands / recombinant fusion protein / Hematology
    anemia indicationsPhase IIIactive LCM
  • OncoACP3OncoACP3 / prostate cancer target / radiopharmaceutical therapeutic/diagnostic agent / Oncology
    prostate canceractive
16assets
  • guselkumabTREMFYA / IL-23 / Monoclonal Antibody / Immunology
    PsoriasisPsoriatic ArthritisCrohn's Disease
  • ibrutinibIMBRUVICA / BTK / Small Molecule / Oncology
    Mantle Cell LymphomaOther B-cell malignancies
  • icotrokinraJNJ-2113 / IL-23R / Targeted Oral Peptide / Immunology
    PsoriasisPsoriatic Arthritis
  • JNJ-4804JNJ-4804 / Co-antibody / Co-antibody Therapy / Immunology
    Ulcerative Colitis
  • nipocalimabIMAAVY / FcRn / Monoclonal Antibody / Immunology / Neuroscience
    Generalized Myasthenia GravisRheumatoid ArthritisWarm Autoimmune Hemolytic Anemia
  • aticaprantKOR antagonist / Small Molecule / Neuroscience
    Adjunctive Treatment for Major Depressive Disorder with Anhedonia
  • RPGR Gene TherapyRPGR / Gene Therapy / Vision / Ophthalmology
    Retinitis Pigmentosa
  • teclistamabTECVAYLI / BCMA x CD3 / Bispecific Antibody / Oncology
    Multiple Myeloma
  • sCD59JNJ-1887 / sCD59 / Gene Therapy / Vision / Ophthalmology
    Geographic Atrophy
  • gemcitabine intravesical delivery systemTAR-200 / INLEXZO / DNA synthesis / Drug Delivery System / Oncology
    High Risk Non-Muscle Invasive Bladder Cancer
  • niraparib / abirateroneAKEEGA / PARP / CYP17 / Small Molecule Combination / Oncology
    Metastatic Castration-Sensitive Prostate CancerMetastatic Castration-Resistant Prostate Cancer
  • daratumumabDARZALEX / CD38 / Monoclonal Antibody / Oncology
    Multiple Myeloma
  • erdafitinibBALVERSA / FGFR / Small Molecule / Oncology
    Urothelial Carcinoma
  • apalutamideERLEADA / Androgen Receptor / Small Molecule / Oncology
    Prostate Cancer
  • ciltacabtagene autoleucelCARVYKTI / BCMA / CAR-T Cell Therapy / Oncology
    Multiple Myeloma
  • Filovirus multivalent vaccineVAC69120 / MVA-BN-Filo / Ebola Virus / Vaccine / Infectious Diseases
    Ebola
16assets
  • NiraparibPARP / Inhibitor / Oncology
    Ovarian Cancer (1st line maintenance)ApprovedOvarian Cancer (Platinum sensitive relapsed maintenance)Approved
  • Glioblastoma (GBM)ApprovedPancreatic Cancer (1st line)Phase 3 / Pivotal
  • RipretinibTKI / Oncology
    Gastrointestinal Stromal Tumors (GIST) (4th line)Approved
  • RepotrectinibROS1 / TRK / Oncology
    ROS1+ NSCLCApprovedNTRK+ solid tumorsApproved
  • Tisotumab vedotinTF / ADC / Oncology
    Cervical Cancer (2nd line+ r/m)Phase 3 / PivotalCervical Cancer (1st line r/m, combo)Phase 2 / POC
  • Zocilurtatug pelitecanZL-1310 / Zoci / DLL3 / ADC / Oncology
    ES-SCLCPhase 3 / PivotalOther NECsPhase 2 / POC
  • ZL-6201LRRC15 / ADC / Oncology
    Sarcoma & CAF+ Solid TumorPhase 1
  • ZL-1311MUC17xCD3 / Oncology
    GI TumorsPre-clinical
  • ZL-1222PD-1/IL-12 / Oncology
    Solid TumorsPre-clinical
  • EfgartigimodFcRn / Immunology
    Generalized Myasthenia Gravis (gMG)ApprovedChronic Inflammatory Demyelinating Polyneuropathy (CIDP)ApprovedMyositisPhase 3 / Pivotal
  • PovetaciceptBAFF/APRIL / Immunology
    IgA NephropathyPhase 3 / PivotalPrimary Membranous NephropathyPhase 2 / POC
  • ElegrobartIGF-1R / Immunology
    TEDPhase 3 / Pivotal
  • ZL-1503IL13/IL31R / Immunology
    Mod-to-Sev ADPhase 1
  • Xanomeline-TrospiumKarXT / Neuroscience
    SchizophreniaApproved
  • OmadacyclineInfectious Disease
    Acute Bacterial Skin and Skin Structure Infection (ABSSSI)ApprovedCommunity-Acquired Bacterial Pneumonia (CABP)Approved
  • Sulbactam-DurlobactamInfectious Disease
    HABP/VABP caused by Susceptible Isolates of Acinetobacter Baumannii-calcoaceticus ComplexApproved
15assets
14assets
  • BotensilimabAGEN1181 / Anti-CTLA-4 / Fc-enhanced anti-CTLA-4 antibody / Oncology
    Non-MSI-H 4L+ metastatic colorectal cancerPhase 3Sites activeNon-MSI-H metastatic colorectal cancer NLMPhase 2Enrollment completePD-1 R/R melanomaPhase 2
  • BalstilimabAGEN2034 / Anti-PD-1 / PD-1 inhibitor antibody / Oncology
    MSS metastatic colorectal cancer in combination with botensilimabPhase 3Sites activePD-1 R/R melanoma in combination with botensilimabPhase 2
  • AGEN2373AGEN2373 / CD137 / CD137 agonist antibody / Oncology
    Solid tumorsPhase 1PD-1 R/R melanoma with botensilimabPhase 2
  • AGEN1571AGEN1571 / Anti-ILT-2 / Antibody / Oncology
    Solid tumorsPhase 1
  • AGEN1777AGEN1777 / Anti-TIGIT x CD96 / Bispecific antibody / Oncology
    Solid tumors with balstilimabPhase 1
  • AGEN1423AGEN1423 / Anti-CD73 x TGFB / Bispecific antibody / Oncology
    Solid tumorsPhase 1
  • INCAGN1876INCAGN1876 / Anti-GITR / Antibody / Oncology
    Solid tumorsPhase 1
  • AGEN1949AGEN1949 / OX40 / OX40 agonist antibody / Oncology
    Solid tumorsPhase 1
  • MK-4830MK-4830 / ILT4 / Anti-ILT4 antagonist antibody / Oncology
    Neoadjuvant ovarian cancerPhase 2Ongoing
  • INCAGN2390INCAGN2390 / TIM-3 / Anti-TIM-3 antagonist antibody / Oncology
    Melanoma and SCCHN in combinationIn development
  • INCAGN2385INCAGN2385 / LAG-3 / Anti-LAG-3 antagonist antibody / Oncology
    Melanoma and SCCHN in combinationIn development
  • CR6086 + BalstilimabCR6086; AGEN2034 / EP4 + PD-1 / EP4 antagonist plus PD-1 antagonist / Oncology
    pMMR-MSS colorectal cancerPhase 1/2Ongoing
  • NLM001 + ZalifrelimabNLM-001 / Hedgehog + CTLA-4 / Hedgehog inhibitor plus CTLA-4 antagonist / Oncology
    Advanced pancreatic cancerPhase 1/2Ongoing
  • OBT076 + BalstilimabOBT076; AGEN2034 / CD205 + PD-1 / Antibody-drug conjugate plus PD-1 antibody / Oncology
    Solid tumorsOngoing
14assets
  • High-concentration, low-volume adalimumabAVT02 / HUMIRA® / Biosimilar / Immunology
    Biosimilar to HUMIRA®ApprovedApprovedU.S.Europe (EEA, UK, Switzerland, Balkans)CanadaAustraliaSaudi ArabiaIsraelEgyptSouth AfricaMoroccoPeruBolivia
  • ustekinumabAVT04 / STELARA® / Biosimilar / Immunology
    Biosimilar to STELARA®ApprovedApprovedU.S.JapanCanadaEU/EEA
  • denosumabAVT03 / PROLIA®/XGEVA® / Biosimilar / Bone Disease
    Biosimilar to PROLIA®/XGEVA®ApprovedApprovedJapanEU/EEA
  • golimumabAVT05 / SIMPONI®/SIMPONI ARIA® / Biosimilar / Immunology
    Biosimilar to SIMPONI®/SIMPONI ARIA®ApprovedApprovedJapanEU/EEAUnited Kingdom
  • afliberceptAVT06 / EYLEA® / Biosimilar / Ophthalmology
    Biosimilar to EYLEA®ApprovedApprovedEU/EEAJapanUnited Kingdom
  • omalizumabAVT23 / XOLAIR® / Biosimilar / Respiratory
  • vedolizumabAVT16 / ENTYVIO® / Biosimilar / Immunology
  • pembrolizumabAVT33 / KEYTRUDA® / Biosimilar / Oncology
  • certolizumab pegolAVT10 / CIMZIA® / Biosimilar / Immunology
  • ixekizumabAVT28 / TALTZ® / Biosimilar / Immunology
  • canakinumabAVT48 / ILARIS® / Biosimilar / Immunology
  • guselkumabAVT41 / TREMFYA® / Biosimilar / Immunology
  • ofatumumabAVT65 / KESIMPTA® / Biosimilar / Immunology
  • dupilumabAVT19 / DUPIXENT® / Biosimilar / Immunology
14assets
  • 177Lu rosopatamab tetraxetanTLX591-Tx / PSMA / Antibody / Urologic Oncology
    Prostate Cancer
  • 177Lu-DOTA-PSMATLX597-Tx / PSMA / Small molecule / Urologic Oncology
    Prostate Cancer
  • 177Lu-girentuximabTLX250-Tx / CAIX / Antibody / Urologic Oncology
    Kidney Cancer / ccRCC
  • 131I-IPATLX101-Tx / LAT / Small molecule / Neuro-oncology
    Brain Cancer / Glioma
  • 90Y-besilesomabTLX66-Tx / CD66 / Antibody / Hematology
    Bone-marrow conditioning
  • 225Ac-RADmAbTLX592-Tx / PSMA / Antibody / Urologic Oncology
    Prostate Cancer
  • 225Ac-girentuximabTLX252-Tx / CAIX / Antibody / Urologic Oncology
    Kidney Cancer
  • UndisclosedTLX400-Tx / FAP / Small molecule / Urologic Oncology
    Bladder Cancer
  • 211At-APATLX102-Tx / LAT / Small molecule / Neuro-oncology
    Brain Cancer
  • -olaratumab conjugateTLX300-Tx / PDGFRα / Antibody / Musculoskeletal Oncology
    Soft Tissue Sarcoma
  • 153Sm-DOTMPTLX090-Tx / Small molecule / Musculoskeletal Oncology
    Bone metastasesPain palliation
  • 68Ga-PSMA-11TLX591-CDx / Illuccix / Gozellix / PSMA / Diagnostic Radiopharmaceutical / Oncology
    Prostate Cancer Imaging
  • 18F-floretyrosineTLX101-CDx / Pixclara / Pixlumi / LAT / Diagnostic Radiopharmaceutical / Neuro-oncology
    Glioma Imaging
  • 89Zr-DFO-girentuximabTLX250-CDx / Zircaix / CAIX / Diagnostic Radiopharmaceutical / Oncology
    Clear Cell Renal Cell Carcinoma (ccRCC) Imaging
13assets
12assets
  • EfgartigimodVYVGART; VYVGART Hytrulo / FcRn / antibody fragment / Autoimmune
    Generalized Myasthenia Gravis (gMG)CommercialChronic Inflammatory Demyelinating Polyneuropathy (CIDP)CommercialPrimary Immune Thrombocytopenia (ITP)CommercialJapan
  • EmpasiprubartC2 / monoclonal antibody / Autoimmune
    Multifocal Motor Neuropathy (MMN)RegistrationalDelayed Graft Function (DGF)Proof of ConceptChronic Inflammatory Demyelinating Polyneuropathy (CIDP)Registrational
  • AdimanebartMuSK / agonist antibody / Neurology
    Congenital Myasthenic Syndromes (CMS)Proof of ConceptSpinal Muscular Atrophy (SMA)Proof of Concept
  • ARGX-109ARGX-109 / IL-6 / antibody / Undisclosed
    UndisclosedPhase 1
  • ARGX-121ARGX-121 / IgA / antibody / Undisclosed
    UndisclosedPhase 1
  • ARGX-213ARGX-213 / FcRn / antibody / Undisclosed
    UndisclosedPhase 1
  • ARGX-118ARGX-118 / Galectin-10 / Airway Inflammation
    Airway InflammationPreclinical
  • ARGX-125ARGX-125 / bispecific antibody / Undisclosed
    UndisclosedPreclinical
  • TSP-101TSP-101 / Fn14 / Undisclosed
    UndisclosedPreclinical
  • LP0145ARGX-112 / IL-22R / SIMPLE Antibody / Dermatology
    Atopic Dermatitispartnered
  • AGMB-101ARGX-114 / MET / HGF-mimetic agonist antibody
  • ABBV-151ARGX-115 / GARP-TGF-beta1 / SIMPLE Antibody / Oncology
    Tumorspartnered
12assets
11assets
  • Taldefgrobep AlfaBHV-2000 / myostatin/activin receptor signaling / recombinant protein; myostatin-activin pathway inhibitor / neuromuscular; metabolic
    Spinal muscular atrophyPhase 3ObesityPhase 1
  • OpakalimBHV-7000 / Kv7.2/Kv7.3 potassium channels / small molecule Kv7 activator / neurology; psychiatry
    Focal epilepsyPhase 3Generalized epilepsyPhase 3Major depressive disorderPhase 2
  • TRPM3 AntagonistBHV-2100 / TRPM3 / oral small molecule antagonist / pain
    Pain disordersPhase 2
  • TYK2/JAK1 InhibitorBHV-8000 / TYK2/JAK1 / brain-penetrant small molecule inhibitor / neuroinflammation
    Parkinson's diseasePhase 3Prevention of amyloid therapy induced ARIAPhase 1Alzheimer's diseasePhase 1
  • IgG DegraderBHV-1300 / IgG / MoDE extracellular protein degrader / immunology
    Graves' disease / rheumatoid arthritisPhase 1
  • IgG DegraderBHV-1310 / IgG / MoDE extracellular protein degrader / immunology
    Myasthenia gravisPreclinical
  • Gd-IgA1 TRAP DegraderBHV-1400 / galactose-deficient IgA1 / TRAP extracellular protein degrader / immunology; renal
    IgA nephropathyPhase 1
  • β1AR Autoantibody DegraderBHV-1600 / β1AR autoantibodies / bifunctional MoDE degrader / cardiovascular
    Peripartum cardiomyopathyPhase 1
  • Trop2 ADC +/- PD1BHV-1510 / TROP2 / antibody-drug conjugate / oncology
    Advanced or metastatic epithelial tumorsPhase 1
  • FGFR3 ADCBHV-1530 / FGFR3 / antibody-drug conjugate / oncology
    Urothelial cancer and other tumorsPhase 1
  • CD30 ADCBHV-1500 / CD30 / antibody-drug conjugate / oncology
    Hodgkin lymphomaPreclinical
11assets
  • LecanemabLEQEMBI / Aβ / mAb / Neurology
    Preclinical Alzheimer's diseasePhase 3Early Alzheimer's disease, SC-AI initiationRegulatory review; FDA PDUFA May 24, 2026U.S.
  • DiranersenBIIB080 / tau / ASO / Neurology
    Early Alzheimer's diseasePhase 2PoC readout reported May 14, 2026
  • BIIB091BIIB091 / BTK / peripheral BTK inhibitor / Neurology
    Multiple sclerosisPhase 2
  • Dapirolizumab pegolCD40L / pegylated antibody fragment / Immunology
    Systemic lupus erythematosusPhase 3
  • LitifilimabBDCA2 / mAb / Immunology
    Systemic lupus erythematosusPhase 3Cutaneous lupus erythematosusPhase 3FDA Breakthrough Therapy DesignationU.S.
  • BIIB142BIIB142 / IRAK4 / degrader / Immunology
    Autoimmune diseasePhase 1
  • FelzartamabCD38 / mAb / Rare disease
    Antibody-mediated rejectionPhase 3IgA nephropathyPhase 3Primary membranous nephropathyPhase 3
  • OmaveloxoloneSKYCLARYS / Nrf2 / activator / Rare disease
    Pediatric Friedreich ataxiaPhase 3
  • SalanersenBIIB115 / SMN / ASO / Rare disease
    Spinal muscular atrophyPhase 3
  • ZorevunersenSCN1A / ASO / Rare disease
    Dravet syndromePhase 3
  • Nusinersen high doseSPINRAZA / SMN2 / splice modulator / Rare disease
    Spinal muscular atrophyApproved in U.S.; under review in E.U.U.S.E.U.
11assets
  • tividenofusp alfa-eknmDNL310; ETV:IDS / AVLAYAH / IDS enzyme replacement; TfR TransportVehicle-enabled delivery / Enzyme TransportVehicle biologic / Lysosomal storage disorders
    Hunter syndrome (MPS II)Approved; Phase 2/3 confirmatory study ongoingU.S. accelerated approval; global Phase 2/3 COMPASS ongoingUSGlobal
  • DNL126ETV:SGSH / SGSH enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disorders
    Sanfilippo syndrome Type A (MPS IIIA)Phase 1/2; Phase 3 plannedOngoing Phase 1/2; global Phase 3 confirmatory study plannedGlobal
  • DNL593TAK-594/DNL593; PTV:PGRN / Progranulin replacement / Protein TransportVehicle biologic / Neurodegenerative disease
    GRN-related frontotemporal dementia (FTD-GRN)Phase 1/2Ongoing; Denali received notice that Takeda will terminate collaboration, with Denali planning to continue developmentGlobal
  • DNL628OTV:MAPT / MAPT / tau / Oligonucleotide TransportVehicle therapeutic / Neurodegenerative disease
    Alzheimer's diseasePhase 1bCTA approved; study start-up underway
  • DNL952ETV:GAA / GAA enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disorders
    Pompe diseasePhase 1IND clinical hold lifted; proceeding to Phase 1US
  • BIIB122/DNL151DNL151; BIIB122 / LRRK2 / Small molecule inhibitor / Neurodegenerative disease
    Parkinson's diseasePhase 2bLUMA enrollment completed; readout expected 2026GlobalLRRK2-associated Parkinson's diseasePhase 2aBEACON ongoing
  • eclitasertibSAR443122/DNL758 / RIPK1 / Small molecule inhibitor / Inflammatory disease
    Ulcerative colitisPhase 2Developed by Sanofi; Phase 2 results expected in 1H 2026
  • DNL921ATV:Abeta / Amyloid beta / Antibody TransportVehicle therapeutic / Neurodegenerative disease
    Alzheimer's diseaseIND-enablingPhase 1/1b initiation planned
  • DNL111ETV:GCase / GCase / Enzyme TransportVehicle biologic / Neurodegenerative disease; lysosomal storage disorders
    Parkinson's diseaseIND-enablingIND-enablingGaucher diseaseIND-enablingIND-enabling
  • DNL622ETV:IDUA / IDUA enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disorders
    MPS IIND-enablingIND-enabling
  • DNL422OTV:SNCA / SNCA / alpha-synuclein / Oligonucleotide TransportVehicle therapeutic / Neurodegenerative disease
    Parkinson's diseaseIND-enablingIND-enabling
11assets
  • INCA033989INCA033989 / mutCALR / Hematology
    CALR-mutated essential thrombocythemia (2L)Phase 3Initiation mid-2026USCALR-mutated myelofibrosis (1L)Phase 1Data expected 2H 2026CALR-mutated myelofibrosis (2L)Phase 3Initiation 2H 2026
  • INCB161734INCB161734 / KRAS G12D / Oncology
    1L G12D-mutated pancreatic ductal adenocarcinoma (PDAC)Phase 3Ongoing (in combo with SOC chemo)KRAS G12D-mutated solid tumorsPhase 1Data expected 2H 2026
  • INCA33890INCA33890 / TGFβR2 × PD-1 / bispecific / Oncology
    1L microsatellite-stable colorectal cancer (MSS CRC)Phase 3Ongoing1L MSS PDAC (combo with SOC)Phase 1Data expected 2H 2026
  • INCB123667INCB123667 / CDK2 / Oncology
    Platinum-resistant ovarian cancer (PROC)PivotalOngoing1L maintenance ovarian cancer (CCNE1+)Phase 3Initiation planned 2H 2026
  • povorcitinibJAK1 / small molecule / Inflammation and Autoimmunity
    Moderate-to-severe hidradenitis suppurativa (HS)Phase 3NDA accepted; approval anticipated 1Q 2027 (US), late 2026 (EU)USEUNonsegmental vitiligoPhase 3Positive data; sNDA submission 1H 2027Moderate-to-severe prurigo nodularis (PN)Phase 3Ongoing; topline data 4Q 2026
  • ruxolitinib creamOpzelura / JAK1/JAK2 / topical / Inflammation and Autoimmunity
    Moderate atopic dermatitisApprovedApproval and launch (ex-US)EUMild-to-moderate hidradenitis suppurativa (HS)Phase 3Ongoing; topline data 4Q 2026
  • axatilimabNiktimvo / CSF-1R / Hematology
    1L chronic GVHD (+ ruxolitinib)PivotalData 2H 20261L chronic GVHD (+ steroids)Data early 20283L+ chronic GVHDApprovedApprovedUS
  • INCB160058INCB160058 / JAK2V617F / Hematology
    JAK2 V617F-mutated myeloproliferative neoplasms (MPNs)Phase 1Data 2H 2026 (ASD formulation)
  • tafasitamabMonjuvi / CD19 / Hematology
    1L diffuse large B-cell lymphoma (DLBCL)Phase 3sBLA submission 1H 2026USRelapsed/refractory DLBCLApprovedApprovedUSEUJapan
  • ruxolitinib XRJakafi XR / JAK1/JAK2 / Hematology
    Myelofibrosis, polycythemia vera, chronic GVHDRegistrationFDA resubmissionUS
  • retifanlimabZynyz / PD-1 / Oncology
    Squamous cell carcinoma of the anal canal (SCAC)ApprovedApproved (EC approval noted)USEUMerkel cell carcinomaApprovedApprovedUSEUJapan
11assets
  • B-VECB-VEC / VYJUVEK / Type VII collagen (COL7A1) / Gene Therapy (HSV-1 vector) / Dermatology
    Dystrophic Epidermolysis Bullosa (DEB)CommercialApprovedGlobal
  • KB105KB105 / Transglutaminase 1 (TGM1) / Gene Therapy / Dermatology
    TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)Phase 1/2ActiveGlobal
  • KB104KB104 / Serine Peptidase Inhibitor Kazal Type 5 (SPINK5) / Gene Therapy / Dermatology
    Netherton SyndromePreclinicalActiveGlobal
  • KB407KB407 / Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) / Gene Therapy / Respiratory
    Cystic FibrosisPhase 1ActiveGlobal
  • KB408KB408 / Alpha-1 antitrypsin / Gene Therapy / Respiratory
    Alpha-1 Antitrypsin Deficiency (AATD)PreclinicalActiveGlobal
  • KB707KB707 / IL-12 and IL-2 / Gene Therapy (HSV-1 vector) / Oncology
    Locally advanced or metastatic solid tumor malignanciesPhase 1ActiveGlobal
  • KB301KB301 / Type III collagen / Gene Therapy / Aesthetics
    Aesthetic Skin ConditionsPhase 1/2ActiveGlobal
  • KB302KB302 / Type I collagen / Gene Therapy / Aesthetics
    Aesthetic Skin ConditionsPreclinicalActiveGlobal
  • KB303KB303 / Elastin / Gene Therapy / Aesthetics
    Aesthetic Skin ConditionsPreclinicalActiveGlobal
  • KB304KB304 / Type III collagen and Elastin / Gene Therapy / Aesthetics
    Aesthetic Skin ConditionsPreclinicalActiveGlobal
  • KB305KB305 / Type IV collagen / Gene Therapy / Aesthetics
    Aesthetic Skin ConditionsPreclinicalActiveGlobal
11assets
  • amlitelimabOX40L / Monoclonal antibody / Immunology
    Atopic dermatitisPhase 3ActiveGlobal
  • tolebrutinibBTK / Small molecule / Neurology
    Multiple sclerosisRegistrationActiveGlobalUS
  • lunsekimigBiologic / Immunology and Inflammation
    Asthma / Respiratory diseasesPhase 2ActiveGlobal
  • rilzabrutinibBTK / Small molecule / Rare Blood Disorders / Immunology
    Sickle cell diseasePhase 2/3ActiveGlobalIgG4-related diseasePhase 3ActiveGlobalAsthmaPhase 2ActiveGlobal
  • venglustatSmall molecule / Rare Diseases
    Gaucher disease type 3 (GD3)Phase 3ActiveGlobal
  • frexalimabCD40L / Monoclonal antibody / Immunology
    Multiple sclerosisPhase 3ActiveGlobal
  • SAR441566SAR441566 / TNFR1si / Small molecule / Immunology
    Immunological diseasesPhase 2ActiveGlobal
  • duvakitugTL1A / Monoclonal antibody / Immunology
    Ulcerative colitisPhase 2ActiveGlobalCrohn's diseasePhase 2ActiveGlobal
  • itepekimabIL-33 / Monoclonal antibody / Immunology and Inflammation
    COPDPhase 3ActiveGlobal
  • isatuximabSarclisa / CD38 / Monoclonal antibody / Oncology
    Multiple myeloma (subcutaneous)RegistrationActiveEU
  • acoziboroleSmall molecule / Infectious Diseases
    Sleeping sicknessRegistrationActiveAfricaEU
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  • Ozuriftamab vedotinBA3021 / ROR2 / CAB-ROR2-ADC / Oncology
    OPSCCPhase 3
  • Mecbotamab vedotinBA3011 / AXL / CAB-AXL-ADC / Oncology
    Sarcoma (soft tissue and bone)Phase 2mKRAS NSCLCPhase 2
  • EvalstotugBA3071 / CTLA-4 / CAB-CTLA-4 antibody / Oncology
    Unresectable and/or metastatic cutaneous melanomaPhase 2
  • BA3182BA3182 / EpCAM x CD3 / Dual CAB T cell engager / Oncology
    AdenocarcinomaPhase 1ongoing
  • BA3361BA3361 / Nectin-4 / CAB-Nectin4-ADC / Oncology
    Multiple tumor typesPreclinicalIND-approved
  • BA3151BA3151 / B7-H4 / CAB-B7H4-ADC / Oncology
    Breast cancerPreclinical
  • BA3142BA3142 / B7-H3 x CD3 / Dual CAB TCE / Oncology
    Multiple tumor typesPreclinicalIND-ready
  • BA3311BA3311 / EGFR x CD3 / TCE / Oncology
    Multiple tumor typesPreclinical
  • BA3241BA3241 / Trop2 x CD3 / Dual CAB TCE / Oncology
    Epidermoid cancerPreclinical
  • BA3362BA3362 / Nectin4 x CD3 / Dual CAB TCE / Oncology
10assets
  • Relacorilantglucocorticoid receptor / selective cortisol modulator / Endocrinology
    Hypercortisolism (GRACE)Phase IIIFiledHypercortisolism associated with adrenal adenoma or hyperplasia (GRADIENT)Phase IIIFiledLong-term safety extensionPhase IIFiled
  • KorlymKorlym / glucocorticoid receptor / cortisol modulator / Endocrinology
    Prevalence and treatment of hypercortisolism in difficult-to-control type 2 diabetes (CATALYST)Phase IV
  • LifyorliLifyorli / glucocorticoid receptor / selective cortisol modulator / Oncology
    Platinum-resistant ovarian cancer (ROSELLA)Phase IIIApproved
  • Relacorilant + Nab-paclitaxel + Bevacizumabglucocorticoid receptor / selective cortisol modulator combination / Oncology
    Platinum-resistant ovarian cancer (BELLA Arm A)Phase IIPlatinum-sensitive ovarian cancer (BELLA Arm B)Phase III
  • Relacorilant + Nab-paclitaxelglucocorticoid receptor / selective cortisol modulator combination / Oncology
    Endometrial cancer (BELLA Arm C)Phase IICervical cancer (STELLA)Phase II
  • Relacorilant + Nab-paclitaxel + Gemcitabineglucocorticoid receptor / selective cortisol modulator combination / Oncology
    Pancreatic cancer (TRIDENT)Phase II
  • Nenocorilant + Nivolumabglucocorticoid receptor / selective cortisol modulator combination / Oncology
    Solid tumors (SYNERGY)Phase II
  • Relacorilant + Enzalutamideglucocorticoid receptor / selective cortisol modulator combination / Oncology
    Prostate cancerPhase II
  • Dazucorilantglucocorticoid receptor / selective cortisol modulator / Neurology
    ALS (DAZALS)Phase II
  • Miricorilantglucocorticoid receptor / selective cortisol modulator / Metabolism
    MASH (MONARCH)Phase II
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  • FruquintinibHMPL-013 / ELUNATE / FRUZAQLA / VEGFR 1/2/3 / Small molecule / Oncology
    Colorectal cancerApproved/MarketedApprovedChinaUSEuropeJapanGastric cancerPhase IIIActiveGlobalOther solid tumors (combination studies)ClinicalActiveGlobal
  • SavolitinibHMPL-504 / AZD6094 / ORPATHYS / c-MET / Small molecule / Oncology
    Non-small cell lung cancer (MET exon 14)Approved/MarketedApprovedChinaNSCLC combinations (with immunotherapy/targeted therapy)Phase IIIActiveGlobal
  • SurufatinibHMPL-012 / SULANDA / VEGFR/FGFR/CSF-1R / Small molecule / Oncology
    Neuroendocrine tumorsApproved/MarketedApprovedChina
  • SovleplenibHMPL-523 / Syk / Small molecule / Hematology/Immunology
    Immune thrombocytopenia (ITP)NDA/Regulatory reviewUnder regulatory reviewChina
  • FanregratinibHMPL-453 / FGFR / Small molecule / Oncology
    Cholangiocarcinoma (FGFR2 fusion)NDA/Regulatory reviewUnder regulatory reviewChina
  • HMPL-760HMPL-760 / BTK (non-covalent) / Small molecule / Oncology/Hematology
    Relapsed/refractory diffuse large B-cell lymphoma (DLBCL)Phase IIIInitiatedChina
  • HMPL-A251HMPL-A251 / HER2 (PI3K/PIKK payload) / Antibody-Targeted Therapy Conjugate (ATTC) / Oncology
    HER2-positive/HER2-low solid tumorsPhase IActiveChinaUS
  • HMPL-A580HMPL-A580 / EGFR (PI3K/PIKK payload) / Antibody-Targeted Therapy Conjugate (ATTC) / Oncology
    Solid tumorsPhase IInitiatedGlobal
  • HMPL-A830HMPL-A830 / ATTC (undisclosed) / Antibody-Targeted Therapy Conjugate (ATTC) / Oncology
    Solid tumorsPreclinical/Phase I plannedPlannedGlobal
  • TazemetostatTAZVERIK / EZH2 / Small molecule / Oncology
    Epithelioid sarcoma / follicular lymphomaLicensed/CommercialLicensed product updateChina
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  • BerobenatidePF-06954522 / GLP-1/GIP / Small Molecule / Internal Medicine
    Chronic Weight ManagementPhase 2bOngoingGlobal
  • elranatamabPF-06863135 / ELREXFIO / BCMA x CD3 / Bispecific Antibody / Oncology
    Double-class Exposed Relapsed / Refractory Multiple MyelomaPhase 3OngoingGlobal
  • ritlecitinibPF-06651600 / LITFULO / JAK3/TEC / Small Molecule / Inflammation and Immunology
    VitiligoPhase 3OngoingGlobalModerate Alopecia AreataPhase 3OngoingGlobal
  • marstacimabPF-06741086 / HYMPAVZI / TFPI / Monoclonal Antibody / Rare Disease
    Hemophilia A/B with InhibitorsRegistrationOngoingGlobal
  • MevrometostatPF-06821497 / EZH2 / Small Molecule / Oncology
    1-2L Metastatic Castration-resistant Prostate Cancer Post-abirateronePhase 3OngoingGlobal
  • Sigvotatug vedotinPF-08046047 / Integrin beta-6 / Antibody-Drug Conjugate (ADC) / Oncology
    Non-Small Cell Lung Cancer (NSCLC)Phase 3OngoingGlobal
  • VepdegestrantARV-471 / Estrogen Receptor (ER) / PROTAC Protein Degrader / Oncology
    ER+/HER2- Metastatic Breast CancerPhase 3OngoingGlobal
  • PCV25PF-07872412 / Pneumococcus / Vaccine / Vaccines
    Pneumococcal InfectionPhase 3OngoingGlobal
  • enfortumab vedotinPADCEV / Nectin-4 / Antibody-Drug Conjugate (ADC) / Oncology
    Muscle-invasive Bladder Cancer (Bladder Sparing)Phase 3OngoingGlobal
  • Lyme Disease Vaccine CandidatePF-07307405 / Borrelia burgdorferi OspA / Vaccine / Vaccines
    Lyme Disease InfectionPhase 3OngoingGlobal
9assets
  • ADG126ADG126 / muzastotug / CTLA-4 / fully human anti-CTLA-4 SAFEbody / Oncology
    advanced/metastatic solid tumorsPhase 1b/2dose expansion ongoing in combination with anti-PD-1 therapies
  • ADG116ADG116 / CTLA-4 / fully human anti-CTLA-4 NEObody / Oncology
    advanced/metastatic solid tumorsPhase 1b/2ongoing
  • ADG206ADG206 / CD137 / masked IgG1 Fc-engineered anti-CD137 POWERbody / Oncology
    advanced/metastatic tumorsPhase 1ongoing
  • ADG106ADG106 / CD137 / fully human ligand-blocking agonistic anti-CD137 IgG4 mAb NEObody / Oncology
    advanced/metastatic solid tumors and/or relapsed/refractory non-Hodgkin's lymphomaPhase 1b/2ongoing
  • ADG153ADG153 / CD47 / masked anti-CD47 IgG1 SAFEbody / Oncology
    hematologic and solid tumorsIND-enablingpreclinical
  • ADG138ADG138 / HER2 x CD3 / HER2xCD3 POWERbody T-cell engager / Oncology
    HER2-expressing solid tumorsIND-enablingpreclinical
  • ADG152ADG152 / CD20 x CD3 / bispecific CD20xCD3 T-cell engager POWERbody / Oncology
    B-cell malignanciesIND-enablingpreclinical
  • ADG104ADG104 / PD-L1 / antibody / Oncology
    cancerPhase 2in development by Sanjin
  • ADG125ADG125 / CSF-1R / antibody / Oncology
    cancerPhase 1in development by Dragon Boat Biopharmaceutical
9assets
  • CASGEVYexa-cel / CASGEVY / BCL11A erythroid-specific enhancer / Non-viral ex vivo CRISPR/Cas9 gene-edited autologous cell therapy / Hemoglobinopathies
    Sickle cell disease, ages 5-11Regulatory submissions expected H1 2026Active pediatric expansionGlobalTransfusion-dependent beta thalassemia, ages 5-11Regulatory submissions expected H1 2026Active pediatric expansionGlobal
  • CTX310CTX310 / ANGPTL3 / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular/metabolic
    Severe hypertriglyceridemiaPhase 1bActive; update expected H2 2026Refractory hypercholesterolemiaPhase 1bActive; update expected H2 2026Heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias or severe hypertriglyceridemiaPhase 1Developed/being evaluated
  • CTX320CTX320 / LPA / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular
    Elevated lipoprotein(a)Clinical trial / dose escalationActive; Lp(a) program update expected in 2026
  • CTX321CTX321 / LPA / Next-generation in vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular
    Elevated lipoprotein(a)IND/CTA-enablingActive
  • CTX460CTX460 / SERPINA1 / In vivo SyNTase gene correction; LNP delivery / Rare disease
    Alpha-1 antitrypsin deficiencyPreclinical; clinical trial expected mid-2026Active
  • CTX340CTX340 / AGT / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular
    Refractory hypertensionIND/CTA-enabling; clinical trial expected H1 2026Active
  • CTX611SRSD107 / Factor XI / FXI mRNA / Long-acting double-stranded siRNA / Thromboembolic/cardiovascular diseases
    Venous thromboembolism prevention after total knee arthroplastyPhase 2Ongoing; top-line data expected H2 2026EuropeAtrial fibrillation, venous thromboembolism, ischemic stroke, cancer-associated thrombosis, chronic kidney disease, peripheral vascular disease, chronic coronary artery diseaseTarget indications disclosed
  • Zugocabtagene geleucelzugo-cel; formerly CTX112 / CD19 / Allogeneic CAR-T cell therapy; CRISPR Cas9 engineered / Autoimmune disease; oncology
    Systemic lupus erythematosusPhase 1Ongoing; update expected H2 2026Systemic sclerosisPhase 1Ongoing; update expected H2 2026Inflammatory myositisPhase 1Ongoing; update expected H2 2026
  • CTX213CTX213 / iPSC-derived allogeneic gene-edited beta islet cell precursor therapy / Regenerative medicine / diabetes
    Type 1 diabetesPreclinical; progressing toward clinicActive
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  • EVT8683BMS-986419 / eIF2b / Neuroscience
    Neurodegeneration
  • CNTX 6016CB2 / Pain
    Pain
  • EVT801VEGFR3 / Oncology
    Oncology
  • EVT401P2X7 / Immunology
    InflammationAutoimmune
  • EVT894Infectious Diseases
    Chikungunya
  • EXS21546A2a / Oncology
    Oncology
  • DSP-11815-HT1A / Neuroscience
    Obsessive Compulsive Disorder (OCD)
  • CT7001CDK7 / Oncology
    Oncology
  • TPM203Immunology
    Pemphigus Vulgaris
9assets
  • DarovasertibIDE196 / PKC / Small Molecule / Oncology
    1L metastatic uveal melanoma (HLA-A*02:01 negative)Phase 2/3Positive topline; NDA initiationGlobalNeoadjuvant primary uveal melanomaPhase 2/3OngoingUS
  • IDE397IDE397 / MAT2A / Small Molecule / Oncology
    MTAP-deleted solid tumorsPhase 1OngoingUS
  • IDE849IDE849 / DLL3 / ADC / Oncology
    DLL3-expressing solid tumors (SCLC, NEC)Phase 1OngoingUS
  • IDE161IDE161 / PARG / Small Molecule / Oncology
    HRD solid tumorsPhase 1RecruitingUS
  • IDE034IDE034 / PTK7/B7H3 / Bispecific ADC / Oncology
    B7H3/PTK7 solid tumors (NSCLC, CRC, breast, ovarian)Phase 1OngoingUS
  • IDE892IDE892 / PRMT5 / Small Molecule / Oncology
    MTAP-deleted solid tumorsPhase 1OngoingUS
  • IDE574IDE574 / KAT6/7 / Small Molecule / Oncology
    Solid tumors (breast, prostate, CRC, lung)Phase 1OngoingUS
  • IDE275IDE275 / Werner Helicase (WRN) / Small Molecule / Oncology
    MSI-high solid tumorsPhase 1OngoingUS
  • IDE705IDE705 / Pol Theta (POLQ) / Small Molecule / Oncology
    HRD solid tumorsPhase 1OngoingUS
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  • BrensocatibBRINSUPRI / DPP1 / Small Molecule / Respiratory / Immunology
    Non-Cystic Fibrosis Bronchiectasis (NCFB)MarketedActiveUSEUHidradenitis SuppurativaPhase 2ActiveGlobal
  • Amikacin Liposome Inhalation SuspensionARIKAYCE / Bacterial 30S ribosomal subunit / Liposomal Inhalation Suspension / Respiratory / Infectious Disease
    Refractory Mycobacterium avium complex (MAC) Lung DiseaseMarketedActiveUSEUJPMAC Lung Disease (Label Expansion)Phase 3ActiveGlobal
  • Treprostinil Palmitil Inhalation PowderTPIP / Prostacyclin receptor / Inhalation Powder / Respiratory / Pulmonary
    Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)Phase 3ActiveGlobalPulmonary Arterial Hypertension (PAH)Phase 3ActiveGlobalProgressive Pulmonary Fibrosis (PPF)Phase 3 PlannedActiveGlobal
  • INS1148INS1148 / Stem Cell Factor SCF248 / Monoclonal Antibody / Respiratory / Immunology
    Interstitial Lung Disease (ILD)Phase 2ActiveGlobalModerate-to-severe asthmaPhase 2ActiveGlobal
  • INS1033INS1033 / Unknown / Unknown / Immunology & Inflammation
    Inflammatory diseasesPreclinicalActiveGlobal
  • Intrathecal gene therapy for DMDDystrophin / Gene Therapy / Neurology / Rare Disease
    Duchenne Muscular Dystrophy (DMD)Phase 1ActiveGlobal
  • INS1202INS1202 / Unknown / Unknown / Neurology / Rare Disease
    Amyotrophic Lateral Sclerosis (ALS)Phase 1ActiveGlobal
  • Stargardt Disease Gene TherapyUnknown / Gene Therapy / Ophthalmology / Rare Disease
    Stargardt DiseasePreclinicalActiveGlobal
  • Next-Gen DPP1 InhibitorsDPP1 / Small Molecule / Immunology & Inflammation
    UndisclosedPreclinicalActiveGlobal
9assets
  • ZanidatamabHER2 / Bispecific Antibody / Oncology
    Biliary Tract Cancers (BTC)Gastroesophageal Adenocarcinoma (GEA)Colorectal Cancer (CRC)
  • LurbinectedinZepzelca / DNA alkylating / Small Molecule / Oncology
    Small Cell Lung Cancer (SCLC)Ewing Sarcoma
  • DordaviproneModeyso / ClpP agonist / DRD2 antagonist / Small Molecule / Oncology
    H3 K27M-mutant diffuse glioma
  • Daunorubicin and cytarabineJZP351 / Vyxeos / Vyxeos Liposomal / Topoisomerase inhibitor / nucleoside metabolic inhibitor / Liposomal Combination / Oncology
    Myelodysplastic Syndromes (MDS)Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)
  • JZP898JZP898 / IFNα2b cytokine pro-drug / Biologic / Oncology
    Solid Tumors
  • JZP3507JZP3507 / ClpP agonist / DRD2 antagonist / Small Molecule / Oncology
    Oncology
  • JZP815JZP815 / pan-RAF / Small Molecule / Oncology
    Solid TumorsHematologic Malignancies
  • CannabidiolEpidyolex / Cannabinoid Receptors / Small Molecule / Neuroscience
    Lennox-Gastaut Syndrome (LGS)Dravet Syndrome (DS)Tuberous Sclerosis Complex (TSC)
  • JZP441JZP441 / Orexin-2 receptor agonist / Small Molecule / Neuroscience
    Narcolepsy Type 1Idiopathic Hypersomnia
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  • Isaralgagene civaparvovecST-920 / alpha-galactosidase A (GLA) / Gene Therapy (AAV) / Metabolic Diseases
    Fabry DiseaseBLA/FilingActiveUS
  • Giroctogene fitelparvovecSB-525 / Factor VIII / Gene Therapy (AAV) / Hematology
    Hemophilia APhase 3ActiveUS
  • ST-503ST-503 / Nav1.7 (SCN9A) / Gene Therapy (Zinc Finger Repressor, AAV9) / Neurology
    Small Fiber Neuropathy (Chronic Neuropathic Pain)Phase 1/2ActiveUS
  • ST-506ST-506 / Prion protein (PRNP) / Gene Therapy (Zinc Finger Repressor, STAC-BBB AAV) / Neurology
    Prion DiseasePreclinicalActiveUS
  • TX200TX200 / HLA-A2 / Cell Therapy (Autologous CAR-Treg) / Transplantation
    Prevention of Immune-Mediated Rejection in HLA-A2 Mismatched Kidney TransplantationPhase 1/2ActiveUSEurope
  • IL23R CAR-TregIL23R / Cell Therapy (CAR-Treg) / Autoimmunity
    Inflammatory Bowel Disease (Crohn's Disease)PreclinicalActiveGlobal
  • Genentech Tau ProgramTau / Gene Therapy (Zinc Finger Epigenetic Regulator) / Neurology
    Tauopathies / Alzheimer's DiseasePreclinicalActiveGlobal
  • Takeda Huntington's ProgramHTT / Gene Therapy (Zinc Finger Epigenetic Regulator) / Neurology
    Huntington's DiseasePreclinicalActiveGlobal
  • Alexion C9ORF72 ProgramC9ORF72 / Gene Therapy (Zinc Finger Transcription Factor) / Neurology
    Amyotrophic Lateral Sclerosis (ALS)PreclinicalActiveGlobalFrontotemporal Lobar Degeneration (FTLD)PreclinicalActiveGlobal
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  • SuzetrigineVX-548 / Journavx / NaV1.8 / Small Molecule / Pain
    Moderate-to-Severe Acute PainPainful Diabetic Peripheral Neuropathy
  • VX-993VX-993 / NaV1.8 / Small Molecule / Pain
    Painful Diabetic Peripheral NeuropathyModerate-to-Severe Acute Pain
  • Vanzacaftor/tezacaftor/deutivacaftorCFTR / Small Molecule / Cystic Fibrosis
    Cystic Fibrosis
  • VX-522VX-522 / CFTR / mRNA / Cystic Fibrosis
    Cystic Fibrosis (non-responsive to modulators)
  • InaxaplinVX-147 / APOL1 / Small Molecule / Kidney Disease
    APOL1-mediated Kidney Disease (AMKD)
  • VX-407VX-407 / PC1 / Small Molecule / Kidney Disease
    Autosomal Dominant Polycystic Kidney Disease (ADPKD)
  • VX-880VX-880 / Islet Cell / Cell Therapy / Type 1 Diabetes
    Type 1 Diabetes
  • VX-264VX-264 / Islet Cell / Cell Therapy / Type 1 Diabetes
    Type 1 Diabetes
  • PovetaciceptALPN-303 / BAFF/APRIL / Biologic / Autoimmune / Kidney Disease
    IgA Nephropathy
9assets
  • XmAb819XmAb819 / ENPP3 x CD3 / Bispecific Antibody / Oncology
    Clear Cell Renal Cell Carcinoma (ccRCC)Phase 1ActiveGlobalColorectal Cancer (CRC)Phase 1ActiveGlobalNon-Small Cell Lung Cancer (NSCLC)Phase 1ActiveGlobal
  • XmAb541XmAb541 / CLDN6 x CD3 / Bispecific Antibody / Oncology
    Ovarian CancerPhase 1ActiveGlobalGerm Cell Tumors (GCT)Phase 1ActiveGlobalSolid TumorsPhase 1ActiveGlobal
  • XmAb808XmAb808 / B7-H3 x CD28 / Bispecific Antibody / Oncology
    Prostate Cancer / Solid TumorsPhase 1ActiveGlobal
  • XmAb942XmAb942 / TL1A / Monoclonal Antibody / Immunology
    Ulcerative Colitis (UC)Phase 2ActiveGlobalInflammatory Bowel Disease (IBD)Phase 2ActiveGlobal
  • XmAb412XmAb412 / TL1A x IL23p19 / Bispecific Antibody / Immunology
    Inflammatory Bowel Disease (IBD)Phase 1ActiveGlobal
  • PlamotamabCD20 x CD3 / Bispecific Antibody / Immunology
    Rheumatoid ArthritisPhase 1ActiveGlobal
  • XmAb657XmAb657 / CD19 x CD3 / Bispecific Antibody / Immunology
    Idiopathic Inflammatory MyositisPhase 1ActiveGlobal
  • ObexelimabCD19 x FcγRIIb / Monoclonal Antibody / Immunology
    Autoimmune DiseasesPhase 3ActiveGlobal
  • TobevibartHepatitis B Virus / Monoclonal Antibody / Infectious Disease
    Chronic Hepatitis B / Hepatitis DPhase 3ActiveGlobal
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  • Insulin AspartAMP-004 / interchangeable biosimilar insulin / Diabetes
  • Recombinant Human InsulinAMP-005 / interchangeable biosimilar insulin / Diabetes
  • AMP-028AMP-028 / biosimilar
  • Intranasal epinephrineAMP-019 / adrenergic receptors / intranasal small molecule / Allergy/Immunology
    Anaphylaxis/allergic reactionsdevelopment
  • AMP-105AMP-105 / peptide / Oncology
    Poorly treated cancersdevelopmentUnited StatesCanada
  • AMP-107AMP-107 / VEGFR / topical eye-drop peptide / Ophthalmology
    Wet age-related macular degenerationdevelopmentUnited StatesCanada
  • AMP-109AMP-109 / peptide-docetaxel conjugate / Oncology
    CancerdevelopmentUnited StatesCanada
  • AMP-110AMP-110 / ACTH receptor pathway / synthetic human corticotropin analog peptide
    Multiple indicationsdevelopmentUnited StatesCanada
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  • PaltusotineCRN00808 / PALSONIFY / SST2 / oral nonpeptide SST2 agonist / Endocrinology; oncology
    Acromegaly (US)ApprovedapprovedUSAcromegaly (EU)Registrationalunder reviewEUCarcinoid syndrome associated with NETsPhase 3clinicalGlobal
  • AtumelnantCRN04894 / MC2R/ACTH receptor / oral nonpeptide ACTH receptor antagonist / Endocrinology
    Congenital adrenal hyperplasia (adult)Phase 3clinicalCongenital adrenal hyperplasia (pediatric)Phase 2/3clinicalACTH-dependent Cushing's syndromePhase 2/3planned
  • CRN09682CRN09682 / SST2 / nonpeptide drug conjugate; MMAE payload / Oncology
    SST2-expressing NETs and other SST2-expressing solid tumorsPhase 1/2clinical
  • PTH AntagonistPTH receptor / oral nonpeptide antagonist / Endocrinology
    Hyperparathyroidism and other diseases of hypercalcemiaPreclinicalIND-enabling
  • TSH AntagonistTSH receptor / oral nonpeptide antagonist / Endocrinology
    Graves' disease; thyroid eye diseasePreclinicalIND-enabling
  • SST3 AgonistSST3 / oral nonpeptide agonist / Nephrology
    Polycystic kidney diseasePreclinicalIND-enabling
  • Oral GLP-1 NonpeptideGLP-1 receptor / oral nonpeptide agonist / Metabolic disease
    Diabetes; obesityPreclinicaldisclosed
  • Oral GIP NonpeptideGIP receptor / oral nonpeptide agonist / Metabolic disease
    Diabetes; obesityPreclinicaldisclosed
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  • CVGBMCVGBM / tumor-associated antigens / unmodified mRNA cancer precision immunotherapy / Oncology
    newly diagnosed surgically resected MGMT-unmethylated glioblastoma or astrocytoma with molecular signature of glioblastomaPhase 1ongoing
  • sqNSCLC shared-antigen cancer precision immunotherapy candidateshared tumor antigens / unmodified mRNA cancer precision immunotherapy / Oncology
    squamous non-small cell lung cancerIND/CTA filedIND and CTA submissions filed Q1 2025
  • CV0601CV0601 / SARS-CoV-2 Omicron BA.4-5 spike antigen / mRNA vaccine / Infectious disease
    COVID-19Phase 2study completed August 2024; GSK assumed full control
  • CV0701CV0701 / SARS-CoV-2 Omicron BA.4-5 and original SARS-CoV-2 antigens / bivalent mRNA vaccine / Infectious disease
    COVID-19Phase 2study completed August 2024; GSK assumed full control
  • seasonal influenza mRNA vaccine candidateinfluenza antigens / multivalent mRNA vaccine / Infectious disease
    seasonal influenzaPhase 2GSK assumed full control
  • avian influenza H5N1 mRNA vaccine candidateH5N1 influenza antigens / mRNA vaccine / Infectious disease
    avian influenza H5N1Phase 2GSK assumed full control
  • COVID-19/influenza combination mRNA vaccine candidateSARS-CoV-2 and influenza antigens / combination mRNA vaccine / Infectious disease
    COVID-19 and influenzaPhase 1/2GSK initiated study November 2024; GSK assumed full control
  • UPEC FimH ferritin nanoparticle mRNA vaccine candidateFimH / mRNA vaccine / Infectious disease
    urinary tract infections caused by uropathogenic E. coliPreclinicallead candidate identified
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  • EVX-01EVX-01 / Personalized neoantigens / Liposomal/peptide personalized cancer vaccine / Cancer
    Advanced melanomaPhase 2
  • EVX-03EVX-03 / Personalized neoantigens and endogenous retrovirus antigens / Targeted DNA personalized cancer vaccine / Cancer
    Solid tumorsPreclinicalCTA/IND ready
  • EVX-04EVX-04 / Tumor-specific antigens derived from endogenous retrovirus elements / DNA off-the-shelf therapeutic cancer vaccine / Cancer
    Acute myeloid leukemiaPreclinicalPlanned first-in-human study
  • EVX-B1EVX-B1 / S. aureus toxins and virulence factors / Protein multi-component prophylactic vaccine / Infectious diseases
    S. aureus skin and soft tissue infectionsPreclinical
  • EVX-B2EVX-B2 / N. gonorrhoeae antigens / Protein multi-component prophylactic vaccine / Infectious diseases
    GonorrheaPreclinical
  • EVX-B2 mRNAEVX-B2 / N. gonorrhoeae antigens / mRNA multi-component prophylactic vaccine / Infectious diseases
    GonorrheaPreclinical
  • EVX-B4EVX-B4 / Group A Streptococcus antigens / Prophylactic vaccine / Infectious diseases
    Group A Streptococcus infectionTarget discovery
  • EVX-V1EVX-V1 / CMV B- and T-cell antigens; pre-fusion glycoprotein B construct / Multi-component prophylactic vaccine / Infectious diseases
    Cytomegalovirus infectionPreclinical
8assets
  • ZanzalintinibXL092 / MET/VEGFR/AXL/MER / Next-generation TKI / Oncology
    Colorectal cancerPivotal / Phase 3STELLAR-303: zanzalintinib + atezolizumab vs. regorafenibNon-clear cell renal cell carcinomaPivotal / Phase 3STELLAR-304: zanzalintinib + nivolumab vs. sunitinibNeuroendocrine tumorsPivotal / Phase 2/3STELLAR-311: zanzalintinib vs. everolimus
  • XL309XL309 / USP1 / USP1 inhibitor / Oncology
    Multiple solid tumorsPhase 1XL309 or XL309 + olaparib
  • XB010XB010 / 5T4 / Next-generation 5T4-MMAE ADC / Oncology
    Multiple solid tumorsPhase 1XB010 or XB010 + pembrolizumab
  • XB628XB628 / PD-L1 + NKG2A / Bispecific antibody / Oncology
    Multiple solid tumorsPhase 1Ongoing clinical study
  • XB371XB371 / Tissue factor / Next-generation TF-TOPOi ADC / Oncology
    Multiple solid tumorsPhase 1Ongoing clinical study
  • ADU-1805ADU-1805 / SIRPα / SIRPα-targeting mAb / Oncology
    Multiple solid tumorsPhase 1ADU-1805 or ADU-1805 + pembrolizumab
  • XB773XB773 / DLL3 / DLL3-TOPOi ADC / Oncology
    Not disclosedDiscovery/PreclinicalDiscovery/Preclinical
  • SSTR2 AgonistSomatostatin receptor subtype 2 / SSTR2 agonist program development candidate / Oncology
    Not disclosedDiscovery/PreclinicalDiscovery/Preclinical
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  • TebentafuspKIMMTRAK / gp100 (HLA-A*02) / ImmTAC (TCR bispecific) / Oncology
    metastatic uveal melanomaApprovedmarketedUSEUadjuvant uveal melanomaPhase 3ongoing (ATOM, EORTC-sponsored)2L+ advanced cutaneous melanomaPhase 3ongoing (TEBE-AM)
  • BrenetafuspIMC-F106C / PRAME (HLA-A*02) / ImmTAC (TCR bispecific) / Oncology
    1L advanced cutaneous melanomaPhase 3ongoing (PRISM-MEL-301)ovarian cancerPhase 1/2ongoing (combinations)NSCLCPhase 1/2ongoing (monotherapy and combinations)
  • IMC-P115CIMC-P115C / PRAME (HLA-A*02 HLE) / ImmTAC (TCR bispecific) / Oncology
    multiple solid tumorsPhase 1ongoing
  • IMC-R117CIMC-R117C / PIWIL1 (HLA-A*02) / ImmTAC (TCR bispecific) / Oncology
    colorectal and GI cancersPhase 1/2ongoing
  • IMC-M113VIMC-M113V / Gag (HLA-A*02) / ImmTAV (TCR bispecific) / Infectious Disease
    HIVPhase 1ongoing
  • IMC-I109VIMC-I109V / Envelope (HLA-A*02) / ImmTAV (TCR bispecific) / Infectious Disease
    HBVclinical development
  • IMC-S118AIIMC-S118AI / PPI x PD1 (HLA-A*02) / ImmTAAI (TCR bispecific) / Autoimmune Disease
    type 1 diabetesPhase 1ongoing
  • IMC-U120AIIMC-U120AI / CD1a x PD1 (non-HLA restricted) / ImmTAAI (TCR bispecific) / Autoimmune Disease
    atopic dermatitisIND-enabling
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  • LacutamabKIR3DL2 / Monoclonal antibody / Oncology
    Sézary syndromeMycosis fungoidesCutaneous T cell lymphomas
  • IPH4502IPH4502 / Nectin-4 / Antibody-drug conjugate (ADC) / Oncology
    Solid tumors
  • MonalizumabNKG2A / Monoclonal antibody / Oncology
    Non-small cell lung cancer (NSCLC)
  • IPH6501IPH6501 / CD20 / NK cell engager (ANKET) / Oncology
    B-cell Non-Hodgkin's Lymphoma
  • IPH6101IPH6101 / CD123 / NK cell engager (ANKET) / Oncology / Autoimmune
    Hematologic malignanciesAutoimmune indications
  • IPH6401IPH6401 / BCMA / NK cell engager (ANKET) / Oncology
    Multiple myeloma
  • IPH5201IPH5201 / CD39 / Monoclonal antibody / Oncology
    Solid tumors
  • IPH5301IPH5301 / CD73 / Monoclonal antibody / Oncology
    Solid tumors
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  • OPGx-LCA5OPGx-LCA5 / LCA5 / AAV Gene Therapy / Ophthalmology
    Leber Congenital Amaurosis Type 5 (LCA5)
  • OPGx-BEST1OPGx-BEST1 / BEST1 / AAV Gene Therapy / Ophthalmology
    Best Vitelliform Macular Dystrophy (BVMD)Autosomal Recessive Bestrophinopathy (ARB)
  • OPGx-RDH12OPGx-RDH12 / RDH12 / AAV Gene Therapy / Ophthalmology
    RDH12-associated Leber Congenital Amaurosis (RDH12-LCA)
  • OPGx-MERTKOPGx-MERTK / MERTK / AAV Gene Therapy / Ophthalmology
    MERTK-associated Retinitis Pigmentosa
  • OPGx-RHOOPGx-RHO / RHO / AAV Gene Therapy / Ophthalmology
    Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
  • OPGx-CNGB1OPGx-CNGB1 / CNGB1 / AAV Gene Therapy / Ophthalmology
    CNGB1-associated Inherited Retinal Diseases
  • OPGx-NMNAT1OPGx-NMNAT1 / NMNAT1 / AAV Gene Therapy / Ophthalmology
    NMNAT1-associated Inherited Retinal Diseases
  • Phentolamine Ophthalmic Solution 0.75%Small Molecule / Ophthalmology
    Pharmacologically induced mydriasisPresbyopiaLow-light visual disturbances following keratorefractive surgery
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  • KT-621KT-621 / STAT6 / Targeted Protein Degrader / Immunology / Respiratory
    Atopic DermatitisPhase 2bOngoingGlobalAsthmaPhase 2bOngoingGlobal
  • KT-579KT-579 / IRF5 / Targeted Protein Degrader / Immunology
    Autoimmune Diseases (Lupus, Rheumatoid Arthritis, IBD)Phase 1PlannedGlobal
  • KT-295KT-295 / TYK2 / Targeted Protein Degrader / Immunology
    Inflammatory Bowel Disease (IBD), PsoriasisPhase 1OngoingGlobal
  • KT-485SAR447971 / IRAK4 / Targeted Protein Degrader / Immunology
    Immuno-inflammatory diseasesPreclinicalOngoingGlobal
  • KT-200KT-200 / CDK2 / Molecular Glue Degrader / Oncology
    Breast Cancer and other Solid TumorsPreclinicalOngoingGlobal
  • KT-333KT-333 / STAT3 / Targeted Protein Degrader / Oncology
    Hematological Malignancies and Solid TumorsPhase 1OngoingGlobal
  • KT-253KT-253 / MDM2 / Targeted Protein Degrader / Oncology
    High-Grade Myeloid Malignancies and Solid TumorsPhase 1OngoingGlobal
  • KT-474SAR444656 / IRAK4 / Targeted Protein Degrader / Immunology
    Hidradenitis Suppurativa (HS), Atopic DermatitisPhase 2bDiscontinuedGlobal
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  • fenebrutinibBTK / Small molecule / Neuroscience
    Multiple Sclerosis
  • petrelintideAmylin / Peptide / Cardiovascular, Renal & Metabolism
    Obesity
  • CT-996CT-996 / GLP-1 / Small molecule / Cardiovascular, Renal & Metabolism
    Obesity
  • giredestrantEstrogen receptor (SERD) / Small molecule / Oncology
    Early and metastatic breast cancer
  • trontinemabAmyloid beta / TfR / Bispecific antibody / Neuroscience
    Alzheimer's Disease
  • NXT007NXT007 / Factor IX/X / Bispecific antibody / Hematology
    Hemophilia A
  • afimkibartBiologic / Immunology
    Immunological disorders
  • RVT-3101RVT-3101 / TL1A / Monoclonal antibody / Immunology
    Inflammatory Bowel Disease
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  • SION-719SION-719 / NBD1 (Nucleotide Binding Domain 1 of CFTR) / Small Molecule / Respiratory / Genetic Diseases
    Cystic FibrosisPhase 2aActive
  • SION-451SION-451 / NBD1 (Nucleotide Binding Domain 1 of CFTR) / Small Molecule / Respiratory / Genetic Diseases
    Cystic FibrosisPhase 1Active
  • SION-638SION-638 / NBD1 (Nucleotide Binding Domain 1 of CFTR) / Small Molecule / Respiratory / Genetic Diseases
    Cystic FibrosisPhase 1Active
  • SION-109SION-109 / ICL4 (Intracellular Loop 4 of CFTR) / Small Molecule / Respiratory / Genetic Diseases
    Cystic FibrosisPhase 1Active
  • GalicaftorABBV-2222 / TMD1-directed CFTR corrector / Small Molecule / Respiratory / Genetic Diseases
    Cystic FibrosisPhase 2Active
  • NavocaftorABBV-3067 / CFTR potentiator / Small Molecule / Respiratory / Genetic Diseases
    Cystic FibrosisPhase 2Active
  • ABBV-2851ABBV-2851 / TMD1-directed CFTR corrector / Small Molecule / Respiratory / Genetic Diseases
    Cystic FibrosisPhase 1Active
  • SION-676SION-676 / TMD1-directed CFTR corrector / Small Molecule / Respiratory / Genetic Diseases
    Cystic FibrosisPreclinicalActive
8assets
  • SRP-1001SRP-1001 / FSHD1 / siRNA / Neuromuscular
    Facioscapulohumeral muscular dystrophy, Type 1 (FSHD1)
  • SRP-1003SRP-1003 / DM1 / siRNA / Neuromuscular
    Myotonic dystrophy, Type 1 (DM1)
  • SRP-1002SRP-1002 / siRNA / Pulmonary
    Idiopathic pulmonary fibrosis (IPF)
  • SRP-1004SRP-1004 / siRNA / Neurology
    Spinocerebellar ataxia type 2 (SCA2)
  • SRP-1005SRP-1005 / siRNA / Neurology
    Huntington's disease (HD)
  • SRP-1007SRP-1007 / siRNA / Neurology
    Spinocerebellar ataxia type 1 (SCA1)
  • SRP-1006SRP-1006 / siRNA / Neurology
    Spinocerebellar ataxia type 3 (SCA3)
  • bidridistrogene xeboparvovecSRP-9003 / LGMD2E/R4 β-sarcoglycan / Gene Therapy / Neuromuscular
    Limb-girdle Muscular Dystrophy
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  • ZanidatamabZW25 / Ziihera / HER2 / Bispecific Antibody / Oncology
    Biliary Tract Cancer (BTC)ApprovedActiveUSEuropeGastroesophageal Adenocarcinoma (GEA)Phase 3ActiveGlobalBreast Cancer (Neoadjuvant and Adjuvant)Phase 2ActiveGlobal
  • ZW191ZW191 / FRα / Antibody-Drug Conjugate (ADC) / Oncology
    Advanced Solid Tumors (Ovarian/Gynecological and Lung/Thoracic)Phase 1ActiveGlobal
  • ZW251ZW251 / GPC3 / Antibody-Drug Conjugate (ADC) / Oncology
    Hepatocellular Carcinoma (HCC) / Solid TumorsPhase 1ActiveGlobal
  • ZW220ZW220 / NaPi2b / Antibody-Drug Conjugate (ADC) / Oncology
    Gynecological and Thoracic CancersPreclinicalActiveGlobal
  • ZW209ZW209 / DLL3 x CD3 x CD28 / Trispecific T-Cell Engager / Oncology
    Thoracic CancersPreclinicalActiveGlobal
  • ZW1528ZW1528 / IL-4Rα x IL-33 / Bispecific Antibody / Autoimmune / Inflammatory Diseases
    Respiratory InflammationPreclinicalActiveGlobal
  • PasritamigJNJ-78278343 / CD3 / Prostate Target / Bispecific Antibody / Oncology
    Solid TumorsClinicalActiveGlobal
  • DS-2243DS-2243 / T-Cell Engager / Bispecific T-Cell Engager / Oncology
    Advanced Solid TumorsPhase 1ActiveGlobal
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  • OlverembatinibHQP1351 / BCR-ABL / small molecule TKI / Oncology
    CML-CP/AP with T315I; CML-CP resistant/intolerant to 1st/2nd gen TKIsMarketedApproved in ChinaChinaCMLPhase 3 registrationalOngoing; POLARIS-2GlobalFirst-line Ph+ ALLPhase 3 registrationalOngoing; POLARIS-1Global
  • LisaftoclaxAPG-2575 / Bcl-2 / small molecule inhibitor / Oncology
    CLL/SLL after systemic therapy including BTK inhibitorsMarketedApproved in China 2025ChinaCLL/SLL after BTK inhibitorsPhase 3 registrationalOngoing; GLORA; +BTK inhibitorGlobalFirst-line CLL/SLLPhase 3 registrationalOngoing; GLORA-2; +acalabrutinibGlobal
  • APG-2449APG-2449 / FAK/ALK/ROS1 / small molecule inhibitor / Oncology
    ALK-positive advanced or locally advanced NSCLC, treatment-naivePhase 3 registrationalOngoing; CDE-clearedChinaNSCLC resistant/intolerant to 2nd generation ALK TKIsPhase 3 registrationalOngoing; CDE-clearedChinaPlatinum-resistant ovarian cancerPhase 1b/2Ongoing; +liposomal doxorubicin
  • AlrizomadlinAPG-115 / MDM2-p53 / small molecule inhibitor / Oncology
    Unresectable/metastatic melanoma and other advanced solid tumorsPhase 1b/2Ongoing; monotherapy or +pembrolizumabUnited StatesAustraliaR/R T-PLL or NHLPhase 2aOngoing; monotherapy or +lisaftoclaxUnited StatesAustraliaSalivary gland cancerPhase 2Collaborative research study
  • PelcitoclaxAPG-1252 / Bcl-2/Bcl-xL / small molecule inhibitor / Oncology
    EGFR-mutant NSCLCPhase 1bOngoing; +osimertinibChinaR/R NHLPhase 1b/2OngoingChinaRecurrent ovarian and endometrial cancersPhase 1Ongoing; +cobimetinib
  • APG-5918APG-5918 / EED/PRC2 / small molecule inhibitor / Oncology; Hematology
    Advanced solid tumors and hematologic malignanciesPhase 1OngoingChinaUnited StatesAnemia-related indicationsPhase 1OngoingChina
  • APG-3288APG-3288 / BTK / PROTAC degrader / Oncology
    R/R B-cell malignanciesPhase 1IND cleared; planned/initiating in 2026United StatesChina
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  • ALTO-207CTC-501 / D3/D2 & 5-HT3 / fixed-dose combination of pramipexole and ondansetron / Neuropsychiatry
    Treatment-resistant depressionPhase 2bactive
  • ALTO-300MT1/2 & 5HT2C / oral small molecule / Neuropsychiatry
    Major depressive disorderPhase 2bactiveUnited States
  • ALTO-100BDNF/neural plasticity / oral small molecule / Neuropsychiatry
    Bipolar depressionPhase 2bactive
  • ALTO-101PDE4 / transdermal PDE4 inhibitor / Neuropsychiatry
    Cognitive impairment associated with schizophreniaPhase 2active
  • ALTO-203Histamine H3 receptor / oral small molecule inverse agonist / Neuropsychiatry
    Major depressive disorder with anhedoniaPhase 2aactive
  • ALTO-202NMDA NR2B/GluN2B / oral small molecule negative allosteric modulator / Neuropsychiatry
    Major depressive disorderactive
  • ALTO-208CTC 413 / D3/D2 & NK-1 / fixed-dose combination of pramipexole and aprepitant / Neurology
    Parkinson's diseaseactive
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  • ARV-102ARV-102 / LRRK2 / oral brain-penetrant PROTAC degrader / Neurology
    Parkinson’s diseasePhase 1/1bunder investigationProgressive supranuclear palsyunder investigation
  • ARV-027ARV-027 / polyQ-AR / oral peripherally restricted PROTAC degrader / Neurology
    Spinal-bulbar muscular atrophyPhase 1/1bunder investigation
  • ARV-806ARV-806 / KRAS G12D / PROTAC degrader / Oncology
    Pancreatic cancerPhase 1/1bunder investigationColorectal cancerPhase 1/1bunder investigationNon-small cell lung cancerPhase 1/1bunder investigation
  • ARV-393ARV-393 / BCL6 / oral PROTAC degrader / Oncology
    Non-Hodgkin lymphoma / B-cell malignanciesPhase 1/1bunder investigation
  • ARV-6723ARV-6723 / HPK1 / oral PROTAC degrader / Oncology
    Advanced solid tumorsPreclinicalunder investigation
  • VepdegestrantARV-471 / ER / oral PROTAC estrogen receptor degrader / Oncology
    ER+/HER2- locally advanced or metastatic breast cancerPhase 3under investigation
  • LuxdegalutamideARV-766; JSB462 / AR / oral PROTAC androgen receptor degrader / Oncology
    Prostate cancerPhase 2under investigation
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  • epcoritamabGEN3013 / EPKINLY/TEPKINLY / CD20xCD3 / Bispecific antibody / Oncology
    Relapsed or refractory diffuse large B-cell lymphoma (DLBCL)ApprovedMarketedUSEUJapanRelapsed or refractory follicular lymphoma (FL)ApprovedMarketedUSEURelapsed or refractory FL in combination with rituximab and lenalidomide (R2)ApprovedMarketedUS
  • rinatabart sesutecanRina-S / Rina-S / FRα (folate receptor alpha) / Antibody-drug conjugate (ADC) / Oncology
    Platinum-resistant ovarian cancerPhase 3ActiveGlobalAdvanced endometrial cancerPhase 1/2ActiveGlobal
  • petosemtamabMCLA-158 / EGFRxLGR5 / Bispecific antibody / Oncology
    First-line head and neck squamous cell carcinoma (HNSCC)Phase 3ActiveGlobalSecond/third-line head and neck squamous cell carcinoma (HNSCC)Phase 3ActiveGlobal
  • daratumumabDARZALEX/DARZALEX FASPRO / CD38 / Monoclonal antibody / Oncology
    Multiple myeloma (multiple lines)ApprovedMarketedGlobal
  • ofatumumabKesimpta/Arzerra / CD20 / Monoclonal antibody / Neurology/Oncology
    Relapsing multiple sclerosis (Kesimpta)ApprovedMarketedGlobalChronic lymphocytic leukemia (Arzerra)ApprovedMarketedUSEU
  • tisotumab vedotinTivdak / Tissue factor / Antibody-drug conjugate (ADC) / Oncology
    Recurrent or metastatic cervical cancerApprovedMarketedUSEUJapan
  • HexaBody-CD38GEN3014 / CD38 / HexaBody monoclonal antibody / Oncology
    Multiple myelomaPhase 1/2ActiveGlobal
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  • AleniglipronGSBR-1290 / GLP-1R / Oral small molecule agonist / Metabolic / Obesity
    Obesity / overweight with weight-related comorbidityPhase 2 (ACCESS / ACCESS II); Phase 3 planned 2H 2026Active; End-of-Phase 2 FDA meeting Q2 2026GlobalType 2 diabetes mellitus with obesity/overweightPhase 2Enrolling (38-week study)Global
  • ACCG-2671ACCG-2671 / Amylin/calcitonin receptor (DACRA) / Oral small molecule agonist / Metabolic / Obesity
    ObesityPhase 1Active (SAD study initiated Dec 2025)Global
  • ACCG-3535ACCG-3535 / Amylin/calcitonin receptor (DACRA) / Oral small molecule agonist / Metabolic / Obesity
    ObesityPreclinical / IND-enablingPhase 1 initiation expected 2H 2026Global
  • ANPA-0073ANPA-0073 / Apelin (APJ) receptor / Oral small molecule biased agonist / Metabolic / Obesity
    Obesity (selective/muscle-sparing weight loss)Phase 2 readyPhase 1 SAD/MAD completedGlobal
  • LTSE-2578LTSE-2578 / LPA1R (lysophosphatidic acid 1 receptor) / Oral small molecule antagonist / Pulmonary / Fibrosis
    Idiopathic pulmonary fibrosis (IPF)Phase 1Phase 1 SAD/MAD completedGlobal
  • GIPR ProgramGIPR (and GLP-1R/GIPR) / Oral small molecule (agonist/antagonist) / Metabolic / Obesity
    Obesity and related diseasesDiscovery / PreclinicalActiveGlobal
  • GCGR ProgramGCGR (and GLP-1R/GCGR) / Oral small molecule agonist / Metabolic / Obesity
    Obesity and related diseasesDiscovery / PreclinicalActiveGlobal
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  • PitolisantWAKIX / histamine-3 receptor (H3R) antagonist/inverse agonist / small molecule / Rare neurological disorders
    Prader-Willi syndromePhase 3USNarcolepsy (GR formulation)Phase 3USNarcolepsy (HD formulation)Phase 3US
  • BP1.15205orexin-2 receptor (OX2R) agonist / small molecule / Sleep/wake disorders
    Narcolepsy and other sleep/wake disordersPhase 1USLatin America
  • EPX-1005-HT (serotonin) receptor agonist / small molecule / Rare epilepsies
    Dravet syndromePhase 3USLennox-Gastaut syndromePhase 3US
  • EPX-200selective 5HT2C agonist / small molecule / Rare epilepsies
    Developmental and epileptic encephalopathiesPreclinicalIND-enablingUS
  • CBS-105cell therapy / Rare neurological disorders
    Treatment-resistant narcolepsyPreclinicalUS
  • HBS-102MCHR1 antagonist / small molecule / Rare neurological disorders
    Prader-Willi syndromePreclinicalUS
  • CBS-104cell therapy / Rare epilepsies
    Refractory epilepsyPreclinicalUS
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  • LifileucelLN-144 / Amtagvi / Polyclonal Neoantigens / Tumor Infiltrating Lymphocyte (TIL) Therapy / Oncology
    MelanomaNon-Small Cell Lung Cancer (NSCLC)
  • TIL CombinationsPolyclonal Neoantigens / Cell Therapy Combination / Oncology
    Solid Tumors
  • PD-1 Selected TILPolyclonal Neoantigens / PD-1+ / Tumor Infiltrating Lymphocyte (TIL) Therapy / Oncology
    Solid Tumors
  • Third Generation (Gen 3) TILPolyclonal Neoantigens / Tumor Infiltrating Lymphocyte (TIL) Therapy / Oncology
    Solid Tumors
  • PBL TherapyPolyclonal Neoantigens / Peripheral Blood Lymphocyte Therapy / Oncology
    Solid Tumors
  • PD-1 Inactivated TILIOV-4001 / PD-1 / Genetically Modified Cell Therapy / Oncology
    Solid Tumors
  • Modified IL-2 Fusion ProteinIOV-3001 / IL-2 Receptor / Antibody Cytokine Engrafted Protein / Oncology
    MelanomaSolid Tumors
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  • LX2006LX2006 / FXN / AAV gene therapy / Cardiovascular
    Friedreich's ataxia (FA) cardiomyopathy
  • LX2020LX2020 / PKP2 / AAV gene therapy / Cardiovascular
    PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM)
  • LX2021LX2021 / Cx43 / AAV gene therapy / Cardiovascular
    DSP (Desmoplakin) cardiomyopathyDilated cardiomyopathy
  • LX2022LX2022 / TNNI3 / AAV gene therapy / Cardiovascular
    Hypertrophic cardiomyopathy (HCM)
  • LX1001LX1001 / APOE2 / AAV gene therapy / Neurology
    APOE4-associated Alzheimer's disease
  • LX1021LX1021 / Christchurch-modified APOE2 / AAV gene therapy / Neurology
    APOE4-associated Alzheimer's disease
  • LX1020LX1020 / APOE2 / APOE4 (miRNA) / AAV gene therapy / Neurology
    APOE4-associated Alzheimer's disease
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  • insulin humanAfrezza / Insulin receptor / Recombinant Protein / Endocrine
    Type 1 and Type 2 Diabetes MellitusMarketedApprovedUSBrazilPediatric Diabetes MellitusPhase 3OngoingUS
  • treprostinilTyvaso DPI / Prostacyclin receptor / Small Molecule / Orphan Lung Diseases
    Pulmonary Arterial Hypertension (PAH)MarketedApprovedUSPulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)MarketedApprovedUS
  • clofazimine inhalation suspensionMNKD-101 / Bacterial DNA / Mycobacterial growth / Small Molecule / Orphan Lung Diseases
    Nontuberculous Mycobacterial (NTM) Lung DiseasePhase 3DiscontinuedUS
  • clofazimine DPIMNKD-102 / Bacterial DNA / Mycobacterial growth / Small Molecule / Orphan Lung Diseases
    Nontuberculous Mycobacterial (NTM) Lung DiseasePreclinicalActiveUS
  • nintedanib DPIMNKD-201 / Tyrosine kinase / Small Molecule / Orphan Lung Diseases
    Idiopathic Pulmonary Fibrosis (IPF)Phase 1CompletedUS
  • dornase alfa DPIMNKD-301 / DNase I / Recombinant Protein / Orphan Lung Diseases
    Cystic FibrosisPreclinicalActiveUS
  • FBM5712 DPIMNKD-501 / ALK-5 kinase / TGF-beta / Small Molecule / Orphan Lung Diseases
    Pulmonary FibrosisPreclinicalActiveUS
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  • SomatrogonhGH-CTP / NGENLA / Growth hormone receptor / Biologic / Endocrinology
    Growth Hormone Deficiency (Pediatric)ApprovedactiveUSEUJapanGlobalGrowth Hormone Deficiency (Adults)Phase 3activeGlobal
  • Calcifediol extended-releaseRayaldee / Rayaldee / Vitamin D receptor / Small Molecule / Nephrology
    Secondary Hyperparathyroidism in Stage 5 Chronic Kidney Disease (Hemodialysis)Phase 2activeUS
  • MDX2004MDX2004 / CD3 x CD28 x 4-1BB / Multispecific Antibody / Oncology
    Advanced CancersPhase 1/2aactiveUS
  • MDX2003MDX2003 / T-cell engager / Multispecific Antibody / Oncology
    LymphomaPhase 1activeUS
  • ModeX COVID-19 Multispecific AntibodySARS-CoV-2 / Multispecific Antibody / Infectious Disease
    COVID-19PreclinicalactiveUS
  • ModeX Influenza Multispecific AntibodyInfluenza virus / Multispecific Antibody / Infectious Disease
    InfluenzaPreclinicalactiveUS
  • Oral GLP-2 AgonistGLP-2 / Peptide / Gastroenterology
    Short Bowel SyndromePreclinicalactiveGlobal
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  • 18F-PivalateRAD 101 / FASN (Fatty acid synthase) / Small Molecule Radiopharmaceutical / Diagnostics
    Brain metastases from solid tumors (Imaging)Phase 2bEnrollingUS
  • 177Lu-RAD202RAD 202 / HER2 / Nanobody Radiotherapeutic / Oncology
    HER2-positive advanced solid tumorsPhase 1ActiveUSGlobal
  • 177Lu-RAD204RAD 204 / PD-L1 / Nanobody Radiotherapeutic / Oncology
    PD-L1 positive advanced solid tumorsPhase 1ActiveUSGlobal
  • 68Ga-TrivehexinRAD 301 / αvβ6-integrin / Peptide Radiopharmaceutical / Diagnostics
    Loco-regional pancreatic cancer (Imaging)Phase 2ActiveGlobal
  • 177Lu-D0301RAD 302 / αvβ6-integrin / Peptide Radiotherapeutic / Oncology
    Solid tumors (PDAC, NSCLC, HNSCC)PreclinicalActiveGlobal
  • BetaBartRV-01 / B7-H3 / Radiotherapeutic / Oncology
    B7H3-expressing solid tumorsPhase 1/2aActiveUS
  • 161Tb-RAD 402RAD 402 / KLK3 (PSA) / Monoclonal Antibody Radiotherapeutic / Oncology
    Advanced prostate cancerPhase 1ActiveAustralia
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  • SGT-003SGT-003 / Gene Therapy / Neuromuscular
    Duchenne muscular dystrophy (INSPIRE)Phase 1/2ActiveGlobalDuchenne muscular dystrophy (IMPACT)Phase 3Activeex-U.S.
  • SGT-212SGT-212 / Gene Therapy / Neuromuscular
    Friedreich's ataxia (FA)Phase 1/2ActiveGlobal
  • SGT-501SGT-501 / Gene Therapy / Cardiac
    RYR2-Mediated CPVTPhase 1/2ActiveGlobalCASQ2-Mediated CPVTPreclinicalActiveGlobal
  • SGT-601SGT-601 / TNNT2 / Gene Therapy / Cardiac
    TNNT2-Mediated Dilated Cardiomyopathy (DCM)PreclinicalActiveGlobal
  • SGT-401SGT-401 / BAG3 / Gene Therapy / Cardiac
    BAG3-Mediated Dilated Cardiomyopathy (DCM)PreclinicalActiveGlobal
  • SGT-701SGT-701 / RBM20 / Gene Therapy / Cardiac
    RBM20-Mediated Dilated Cardiomyopathy (DCM)PreclinicalActiveGlobal
  • Undisclosed Cardiac TargetsGene Therapy / Cardiac
    Genetic cardiomyopathies and channelopathiesDiscoveryActiveGlobal
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  • SPY001SPY001 / α4β7 / Monoclonal antibody / Gastroenterology
    Inflammatory Bowel Disease (IBD)Ulcerative Colitis
  • SPY002SPY002 / TL1A / Monoclonal antibody / Gastroenterology / Rheumatology
    Inflammatory Bowel Disease (IBD)Rheumatoid Arthritis (RA)
  • SPY003SPY003 / IL-23 / Monoclonal antibody / Gastroenterology
    Inflammatory Bowel Disease (IBD)
  • SPY120SPY120 / α4β7 + TL1A / Combination / Gastroenterology
    Inflammatory Bowel Disease (IBD)
  • SPY130SPY130 / α4β7 + IL-23 / Combination / Gastroenterology
    Inflammatory Bowel Disease (IBD)
  • SPY230SPY230 / TL1A + IL-23 / Combination / Gastroenterology
    Inflammatory Bowel Disease (IBD)Ulcerative Colitis
  • SPY072SPY072 / TL1A / Monoclonal antibody / Rheumatology
    Rheumatoid Arthritis (RA)Psoriatic Arthritis (PsA)Axial Spondyloarthritis (axSpA)
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  • VY7523VY-TAU01 / Tau / Monoclonal Antibody / Neurology
    Alzheimer's DiseasePhase 1OngoingUnited States
  • VY1706Tau / AAV Gene Therapy / Neurology
    Alzheimer's DiseaseIND-EnablingOngoingUnited States
  • APOE Gene TherapyAPOE / AAV Gene Therapy / Neurology
    Alzheimer's DiseasePreclinicalOngoing
  • NBIB-'223NBIB-223 / Frataxin (FXN) / AAV Gene Therapy / Neurology
    Friedreich's Ataxia (FA)IND-EnablingOngoing
  • GBA1 Gene TherapyGBA1 / AAV Gene Therapy / Neurology
    Parkinson's Disease and GBA1-mediated diseasesIND-EnablingOngoing
  • Novartis Partnered ProgramsUndisclosed / AAV Gene Therapy / Neurology
    Huntington's Disease, Spinal Muscular Atrophy (SMA), UndisclosedPreclinicalOngoing
  • Alexion Partnered ProgramUndisclosed / AAV Gene Therapy / Neurology
    Undisclosed Neurological DiseasePreclinicalOngoing
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  • RemlifanserinACP-204 / 5-HT2A receptor / inverse agonist / Neurological Disorders
    Alzheimer’s Disease PsychosisPhase 2RecruitingLewy Body Dementia with PsychosisPhase 2
  • ACP-211ACP-211 / selectively deuterated R-norketamine / Neurological Disorders
    Major Depressive DisorderPhase 2
  • ACP-711ACP-711 / GABAA α3 / modulator / Neurological Disorders
    Essential TremorPhase 1
  • ACP-271ACP-271 / GPR88 / agonist / Neurological Disorders; Rare Diseases
    Tardive DyskinesiaIND EnablingHuntington's DiseaseIND Enabling
  • ACP-2591ACP-2591 / cGP analog / Rare Diseases
    Rett Syndrome; Fragile X SyndromePhase 1
  • STOKE ASOantisense oligonucleotide / Rare Diseases
    SYNGAP1Discovery
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  • ACI-35.030JNJ-2056; JNJ-64042056 / phosphorylated Tau (pTau) / active immunotherapy / neurodegenerative diseases
    preclinical Alzheimer's diseasePhase 2bongoing
  • ACI-24.060amyloid beta (Abeta) / active immunotherapy / neurodegenerative diseases
    Alzheimer's diseasePhase 2ongoingAlzheimer's disease in Down syndromePhase 2ongoing
  • ACI-7104.056alpha-synuclein (a-syn) / active immunotherapy / neurodegenerative diseases
    Parkinson's diseasePhase 2ongoing
  • ACI-19764NLRP3 inflammasome / small molecule inhibitor / neuroinflammation
    neuro-inflammationPhase 1ongoing
  • Morphomer TauTau / small molecule / neurodegenerative diseases
    Alzheimer's diseasePreclinicalIND-enabling studies planned
  • Morphomer a-synalpha-synuclein (a-syn) / small molecule / neurodegenerative diseases
    Parkinson's diseasePreclinicalreadiness to initiate IND-enabling studies
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  • AL101PGRN / antibody / Neurodegenerative diseases
    Alzheimer’s diseasePhase 2
  • AL137-ABCAmyloid beta / ABC-enabled antibody / Neurodegenerative diseases
    Alzheimer’s diseasePreclinical
  • AL050-ABCGCase / ABC-enabled enzyme replacement therapy / Neurodegenerative diseases
    Parkinson’s diseasePreclinicalGaucher diseasePreclinicalLewy body dementiaPreclinical
  • AL064-ABCTau / ABC-enabled siRNA / Neurodegenerative diseases
    Alzheimer’s diseaseResearchFrontotemporal dementiaResearch
  • ADP062-ABCAlpha-synuclein / ABC-enabled siRNA / Neurodegenerative diseases
    Parkinson’s diseaseResearch
  • ADP065-ABCNLRP3 / ABC-enabled siRNA / Neurodegenerative diseases
    Alzheimer’s diseaseResearchParkinson’s diseaseResearch
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  • cemacabtagene ansegedleucelcema-cel; ALLO-501A / CD19 / Allogeneic CAR T cell therapy / Hematologic malignancies
    Large B-cell lymphoma, MRD-positive first-line consolidationPivotal Phase 2EnrollingUSCanadaSouth KoreaAustralia
  • ALLO-316ALLO-316 / CD70 / Allogeneic CAR T cell therapy / Solid tumors; hematologic malignancies
    Advanced/metastatic clear cell renal cell carcinomaPhase 1bExpansion cohort enrollment completed; strategic opportunities being exploredCD70+ hematologic malignanciesIND-enablingCD70+ solid tumorsIND-enabling
  • ALLO-329ALLO-329 / CD19/CD70 / Allogeneic dual CAR T cell therapy; CRISPR gene-edited; Dagger technology / Autoimmune disease
    Systemic lupus erythematosus including lupus nephritisPhase 1OngoingIdiopathic inflammatory myopathiesPhase 1OngoingSystemic sclerosisPhase 1Ongoing
  • ALLO-213ALLO-213 / DLL3 / Allogeneic CAR T cell therapy / Solid tumors
    Small cell lung cancerIND-enabling
  • ALLO-182ALLO-182 / Claudin 18.2 / Allogeneic CAR T cell therapy / Solid tumors
    Gastric and pancreatic solid tumorsIND-enabling
  • BCMA/CD70 dual CAR T constructBCMA/CD70 / Allogeneic dual CAR T cell therapy; Dagger technology / Hematologic malignancies
    Multiple myelomaPreclinical
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  • obe-celAUCATZYL / CD19 / autologous CAR T-cell therapy / Hematology/oncology; autoimmune
    r/r adult ALLApprovedFDA/MHRA/EMA approvedUSUKEUPediatric B-ALLPhase 2CATULUSSystemic Lupus ErythematosusPhase 1CARLYSLE
  • AUTO1/22CD19 & CD22 / dual-targeting autologous CAR T-cell therapy / Hematology/oncology
    Pediatric B-ALLPhase 1CARPALL
  • AUTO8BCMA & CD19 / autologous CAR T-cell therapy / Hematology/oncology
    Multiple MyelomaPhase 1MCARTYLight Chain AmyloidosisPhase 1ALARIC
  • AUTO4/5TRBC1/2 / programmed T-cell therapy / Hematology/oncology
    TRBC1/2+ Peripheral TCLPreclinicalLibrA T1
  • AUTO6NGGD2 / autologous CAR T-cell therapy / Solid tumors
    NeuroblastomaPhase 1MAGNETO
  • AUTO9programmed T-cell therapy / Hematology/oncology
    Acute Myeloid LeukemiaPreclinicalTBD
6assets
  • AXS-05AXS-05 / AUVELITY / NMDA receptor; sigma-1 receptor; CYP2D6 / oral small molecule; dextromethorphan-bupropion / CNS
    Smoking cessationPhase 2/3 plannedOn track to initiate in Q2 2026US
  • SolriamfetolSUNOSI / DNRI; TAAR1; 5-HT1A / oral small molecule / CNS
    ADHD, childrenPhase 3 plannedOn track to initiate in Q2 2026USADHD, adolescentsPhase 3 plannedOn track to initiate in Q2 2026USMDD with EDS symptomsPhase 3CLARITY initiatedUS
  • AXS-12AXS-12 / Norepinephrine reuptake inhibitor; cortical dopamine modulator / oral small molecule; reboxetine / CNS
    Cataplexy in narcolepsyNDA submittedFDA filing acceptance decision pendingUS
  • AXS-14AXS-14 / Norepinephrine reuptake inhibitor / oral small molecule; esreboxetine / CNS
    FibromyalgiaPhase 3FORWARD ongoingUS
  • AXS-17AXS-17 / GABA-A receptor α2,3 subtype-selective PAM / oral small molecule / CNS
    EpilepsyPhase 2 trial-enablingUnderwayUS
  • AXS-20AXS-20 / PDE10A / oral small molecule; balipodect / CNS
    SchizophreniaPre-Phase 3Phase 3 trial-enabling activities anticipated in 2026USTourette syndromePlanned evaluationUS
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  • Ristoglogene autogetemcelBEAM-101 / Risto-cel / HBG1/2 promoter; fetal hemoglobin activation / Ex vivo autologous CD34+ HSPC base-edited cell therapy / Hematology
    Sickle cell diseasePhase I/IIOngoing BEACON Phase 1/2 trial; FDA orphan drug and RMAT designationsU.S.
  • ESCAPEBEAM-103 / CD117; HbF edit + CD117 edit-antibody pair / Anti-CD117 monoclonal antibody plus edited cell therapy; in vivo and/or ex vivo / Hematology
    Sickle cell diseasePhase I-EnablingBeta-thalassemiaPhase I-Enabling
  • In vivo HSC editingHbF activation / In vivo LNP base editing / Hematology
    Sickle cell diseaseResearchBeta-thalassemiaResearch
  • BEAM-302BEAM-302 / SERPINA1 E342K/PiZ mutation correction / In vivo LNP base editing / Liver-targeted genetic diseases
    Alpha-1 antitrypsin deficiencyPhase I/IIOngoing Phase 1/2 trial; FDA orphan drug and RMAT designationsU.S.
  • BEAM-301BEAM-301 / G6PC R83C mutation correction / In vivo LNP base editing / Liver-targeted genetic diseases
    Glycogen storage disease IaPhase I/IIOngoing Phase 1/2 trial
  • BEAM-304BEAM-304 / PAH mutation correction / In vivo LNP base editing / Liver-targeted genetic diseases
    PhenylketonuriaPhase I-EnablingPlanned Phase 1/2 trialU.S.
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  • BXCL501BXCL501 / IGALMI / alpha-2 receptor agonist / orally dissolving/sublingual film formulation of dexmedetomidine / Neuroscience
    Acute agitation associated with bipolar disorders or schizophrenia, at homePhase 3Company-sponsored SERENITY programAcute agitation associated with Alzheimer's dementiaPhase 3Company-sponsored TRANQUILITY program; paused subject to fundingOpioid use disorderPhase 2Investigator-sponsored
  • BXCL701BXCL701 / DPP8/9 and FAP / oral innate immune activator / Immuno-oncology
    Small cell neuroendocrine prostate cancerPhase 2Company-sponsored; FDA meeting expectedUSUKSmall cell lung cancerPreclinicalCompany-sponsored; initiate Phase 1b/2 expectedMetastatic pancreatic ductal adenocarcinomaPhase 2Investigator-sponsored; Phase 2 readout expected
  • BXCL502BXCL502 / latrepirdine / Neuroscience
    Chronic agitation in dementiaPreclinicalDevelopment paused due to strategic reprioritization
  • BXCL503BXCL503 / Neuroscience
    Apathy in dementiaPreclinicalDevelopment paused due to strategic reprioritization
  • BXCL504BXCL504 / Neuroscience
    Aggression in dementiaPreclinicalDevelopment paused due to strategic reprioritization
  • BXCL702BXCL702 / DPP inhibitor / novel DPP inhibitor / Immuno-oncology
    Solid tumorsPreclinicalBXCL701 follow-on; candidate nomination expected
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  • AmglidiaAmglidia / glyburide oral suspension / Pediatric endocrinology / rare disease
    Neonatal diabetes mellitusLate-stage development; NDA submission anticipated in 2026In developmentUnited States
  • ET-700ET-700 / extended-release zinc acetate formulation / Rare metabolic disease
    Wilson diseasePilot clinical study / proof-of-conceptFirst patient dosed; topline results expected H2 2026; potential pivotal study in early 2027United States
  • ET-800ET-800 / Rare disease
    UndisclosedLate-stage developmentIn development
  • ZENEO hydrocortisone autoinjectorZENEO / hydrocortisone autoinjector / Endocrinology
    Adrenal insufficiency / adrenal crisis managementLate-stage developmentIn developmentUnited States
  • KHINDIVI revised formulationKHINDIVI / hydrocortisone oral solution / Pediatric endocrinology
    Adrenocortical insufficiency label expansion to younger pediatric patientsBioequivalence study; supplemental NDA plannedStudy initiated; supplement expected in Q3 2026; potential mid-2027 approvalUnited States
  • INCRELEX label expansionINCRELEX / IGF-1 receptor pathway / mecasermin injection / Pediatric endocrinology
    Severe primary IGF-1 deficiency definition harmonization / label expansionPlanned clinical studyFDA feedback requested; study could initiate in H1 2026 if favorableUnited States
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  • FT819FT819 / CD19 / iPSC-derived off-the-shelf CAR T-cell therapy / Autoimmunity
    Systemic Lupus ErythematosusPhase 1RMATUSSystemic Sclerosis (SSc)Phase 1USANCA associated Vasculitis (AAV)Phase 1US
  • FT839FT839 / CD19/CD38/CD20; CD19/CD38/BCMA/GPRC5D / Next-generation CAR T-cell therapy / Autoimmunity; Oncology
    Pan-indication without lympho-conditioning chemotherapyPhase 1USPan-indication (hematologic) without lympho-conditioning chemotherapyPhase 1US
  • FT522FT522 / CD19/CD20 / Cellular immunotherapy / Autoimmunity
    Pan-indication without lympho-conditioning chemotherapyPhase 1US
  • FT825FT825 / HER2/EGFR / iPSC-derived off-the-shelf CAR T-cell therapy / Oncology
    Solid tumorsPhase 1EnrollingUS
  • UndisclosedUndisclosed / Cellular immunotherapy / Oncology
    Solid tumorsPhase 1US
  • FT836FT836 / MICA/B/EGFR/HER2 / Next-generation CAR T-cell therapy / Oncology
    Pan-indication (solid/hematologic) without lympho-conditioning chemotherapyPhase 1US
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  • Symvess (ATEV)Symvess / Acellular Tissue Engineered Vessel / Vascular Disease
    Vascular trauma repairApprovedFDA Approved (Dec 2024)United StatesArteriovenous access for hemodialysisPhase 3V007 met primary endpoints; V012 ongoing; sBLA planned H2 2026United StatesPeripheral artery disease (PAD)Phase 2Completed Phase 2 trials and expanded access; positive limb salvage dataUnited StatesEurope
  • CTEVCoronary Tissue Engineered Vessel / Cardiovascular Disease
    Coronary artery bypass grafting (CABG)PreclinicalPositive NHP data; IND submitted; first-in-human planned H2 2026United States
  • Biovascular Pancreas (BVP)Biovascular Pancreas™ / Bioengineered Organ / Diabetes
    Type 1 diabetesPreclinicalPositive preclinical data; NHP studies planned; first-in-human planned 2027
  • Urinary ConduitAdvanced Tissue Construct / Urology
    Urinary diversion after cystectomyPreclinical
  • Tracheal ReplacementAdvanced Tissue Construct / Otolaryngology
    Tracheal replacementPreclinical
  • Esophageal ReplacementAdvanced Tissue Construct / Gastroenterology
    Esophageal replacementPreclinical
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  • INO-3107INO-3107 / HPV-6 and HPV-11 / DNA immunotherapy / HPV-related rare disease
    Recurrent Respiratory Papillomatosis (RRP) in adultsBLA accepted under accelerated approval, PDUFA Oct 30 2026United States
  • VGX-3100VGX-3100 / HPV-16/18 / DNA immunotherapy / HPV-related diseases
    Cervical dysplasia (HSIL)3Ongoing (partnered)ChinaAnal dysplasia
  • INO-3112INO-3112 / HPV-16/18 / DNA immunotherapy / Oncology
    HPV16/18-positive oropharyngeal squamous cell carcinoma (OPSCC)3Phase 3 planningUnited StatesEurope
  • INO-5401INO-5401 / Tumor-associated antigens / DNA immunotherapy / Oncology
    Glioblastoma (GBM)1Ongoing Phase 1; Phase 2 planning (as INO-5412 w/ Akeso)United StatesPrevention in BRCA1/2 mutation carriers1Ongoing Phase 1United States
  • INO-4201INO-4201 / Ebola virus / DNA medicine / Infectious diseases
    Ebola vaccine booster (to ERVEBO)2Phase 2 planning; Phase 1b completed
  • DMAb SARS-CoV-2SARS-CoV-2 / DNA-encoded monoclonal antibody / Infectious diseases
    SARS-CoV-21Ongoing Phase 1; data expected/reportedUnited States
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  • PemivibartPEMGARDA / SARS-CoV-2 / Monoclonal Antibody / Infectious Diseases
    Pre-exposure prophylaxis (PrEP) of COVID-19
  • VYD2311VYD2311 / SARS-CoV-2 / Monoclonal Antibody / Infectious Diseases
    COVID-19 prevention
  • AdintrevimabSARS-CoV-2 / Monoclonal Antibody / Infectious Diseases
    COVID-19 prevention and treatment
  • Measles mAbMeasles (rubeola) virus / Monoclonal Antibody / Infectious Diseases
    Measles treatment and post-exposure prophylaxis
  • RSV mAbRespiratory Syncytial Virus (RSV) / Monoclonal Antibody / Infectious Diseases
    RSV infection
  • Influenza mAbInfluenza virus / Monoclonal Antibody / Infectious Diseases
    Influenza infection
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  • MP0712MP0712 / DLL3 / Radio-DARPin Therapy / Oncology
    Small Cell Lung Cancer (SCLC)Neuroendocrine Tumors
  • MP0726MP0726 / MSLN / Radio-DARPin Therapy / Oncology
    Ovarian Cancer
  • MP0533MP0533 / CD33 x CD123 x CD70 x CD3 / Multispecific T Cell Engager / Oncology
    Relapsed/Refractory AMLAML/MDS
  • MP0317MP0317 / FAP x CD40 / Tumor-localized CD40 Agonist / Oncology
    Advanced Solid TumorsCholangiocarcinoma
  • MP0632MP0632 / CD2 x CD3 x MSLN x EpCAM / Switch-DARPin T Cell Engager / Oncology
    MSLN/EpCAM Expressing Solid TumorsOvarian Cancer
  • MP0621MP0621 / cKIT x CD16a x CD47 / Switch-DARPin / Hematology
    Conditioning for Hematopoietic Stem Cell Transplantation
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  • DA-1726DA-1726 / GLP1R / GCGR (Glucagon-like peptide-1 receptor and glucagon receptor) / Oxyntomodulin (OXM) analogue / Cardiometabolic Diseases
    ObesityPhase 1ActiveGlobalMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 1ActiveGlobal
  • VanoglipelDA-1241 / GPR119 (G-protein-coupled receptor 119) / Small Molecule / Cardiometabolic Diseases
    Metabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 2aActiveGlobalType 2 Diabetes (T2D)Phase 1CompletedGlobal
  • ANA001ANA001 / Unknown / Oral Niclosamide Formulation / Infectious Diseases
    Moderate COVID-19Phase 2LegacyUS
  • NB-01NB-01 / Unknown / Unknown / Neurology
    Painful Diabetic NeuropathyUnknownLegacyGlobal
  • NB-02NB-02 / Unknown / Unknown / Neurology
    Cognitive Impairment / Neurodegenerative DiseasesUnknownLegacyGlobal
  • GemcabeneUnknown / Small Molecule / Cardiovascular / Infectious Diseases
    DyslipidemiaUnknownLegacyGlobalAcute COVID-19UnknownLegacyGlobal
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  • OST-HER2OST31-164 / HER2 / Bioengineered Listeria monocytogenes (Immunotherapy) / Oncology
    Osteosarcoma (Pulmonary Metastatic)Phase 2bActiveGlobalBreast CancerPhase 1ActiveGlobalCanine OsteosarcomaApprovedMarketedUS
  • OST-AXALAXAL / ADXS-HPV / HPV / Listeria-based Immunotherapy / Oncology
    HPV-associated cancersPhase 3ActiveGlobal
  • OST-503ADXS-503 / KRAS G12 position-related antigens / Listeria-based Immunotherapy / Oncology
    Non-Small Cell Lung Cancer (NSCLC)Phase 2ActiveGlobalGlioblastoma (GBM)Phase 2ActiveGlobalPancreatic CancerPreclinicalActiveGlobal
  • OST-504OST-PSA / ADXS-504 / ADXS31142 / PSA / Listeria-based Immunotherapy / Oncology
    Prostate Cancer (Castration-Resistant)Phase 1bActiveGlobal
  • OST-tADCVarious / Tunable Antibody-Drug Conjugate (ADC) / Oncology
    Ovarian CancerPreclinicalActiveGlobalBreast CancerPreclinicalActiveGlobal
  • OST-HOT constructsShared hotspot mutations and cancer-testes antigens / Listeria-based Immunotherapy / Oncology
    Solid TumorsPreclinicalActiveGlobal
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  • AX-0810AX-0810 / NTCP (SLC10A1) / RNA Editing Oligonucleotide (Axiomer EON) / Liver
    Biliary AtresiaPrimary Sclerosing Cholangitis
  • AX-0811AX-0811 / NTCP / RNA Editing Oligonucleotide (Axiomer EON) / Liver
    Cholestatic Diseases
  • AX-0422AX-0422 / IDUA / RNA Editing Oligonucleotide (Axiomer EON) / Metabolic / Rare Disease
    Hurler Syndrome
  • AX-2402AX-2402 / MECP2 (R270X) / RNA Editing Oligonucleotide (Axiomer EON) / CNS / Rare Disease
    Rett Syndrome
  • AX-2911AX-2911 / PNPLA3 / RNA Editing Oligonucleotide (Axiomer EON) / Liver / Metabolic
    MASH
  • AX-1412AX-1412 / B4GALT1 / RNA Editing Oligonucleotide (Axiomer EON) / Cardiovascular
    Cardiovascular Disease
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  • rivunatpagene miziparvovecUX701 / ATP7B / AAV9 gene therapy / Inborn Errors of Metabolism
    Wilson disease
  • rebisufligene etisparvovecUX111 / AAV gene therapy / Neurogenetic
    Sanfilippo syndrome type A (MPS IIIA)
  • avalotcagene ontaparvovecDTX301 / OTC / AAV8 gene therapy / Inborn Errors of Metabolism
    Ornithine transcarbamylase (OTC) deficiency
  • DTX401DTX401 / AAV gene therapy / Inborn Errors of Metabolism
    Glycogen storage disease type Ia (GSDIa)
  • GTX-102GTX-102 / UBE3A / Antisense oligonucleotide (ASO) / Neurogenetic
    Angelman Syndrome
  • setrusumabUX143 / Biologic / Bone-Endocrine
    Osteogenesis imperfecta
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  • DaraxonrasibRMC-6236 / RAS(ON) multi-selective / Small Molecule / Oncology
    Pancreatic Ductal Adenocarcinoma (PDAC)Non-Small Cell Lung Cancer (NSCLC)Colorectal Cancer (CRC)
  • ZoldonrasibRMC-9805 / RAS(ON) G12D / Small Molecule / Oncology
    Non-Small Cell Lung Cancer (NSCLC)Solid Tumors
  • ElironrasibRMC-6291 / RAS(ON) G12C / Small Molecule / Oncology
    KRAS-G12C mutant cancersNon-Small Cell Lung Cancer (NSCLC)Solid Tumors
  • RMC-5127RMC-5127 / RAS(ON) G12V / Small Molecule / Oncology
    RAS-G12V mutant cancers
  • RMC-0708RMC-0708 / RAS(ON) Q61H / Small Molecule / Oncology
    RAS-Q61H mutant cancers
  • RMC-8839RMC-8839 / RAS(ON) G13C / Small Molecule / Oncology
    RAS-G13C mutant cancers
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  • SGR-1505SGR-1505 / MALT1 / Small Molecule / Oncology
    Relapsed/Refractory B-cell MalignanciesPhase 1Active
  • SGR-3515SGR-3515 / PKMYT1 / Wee1 / Small Molecule / Oncology
    Advanced Solid TumorsPhase 1Active
  • SGR-4174SGR-4174 / SOS1 / Small Molecule / Oncology
    KRAS-driven CancersPreclinicalActive
  • SGR-2921SGR-2921 / CDC7 / Small Molecule / Oncology
    Acute Myeloid Leukemia (AML) / Myelodysplastic Syndrome (MDS)Phase 1Discontinued
  • ACCG-2671ACCG-2671 / Calcitonin Receptor (DACRA) / Small Molecule / Metabolic
    ObesityPhase 1Active
  • AJ1-11095AJ1-11095 / JAK2 / Small Molecule / Hematology
    MyelofibrosisPreclinicalActiveUS
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  • DivesiranSLN124 / TMPRSS6 / siRNA / Hematologic Conditions
    Polycythemia VeraOther Blood Disorders
  • ZerlasiranSLN360 / LPA / siRNA / Cardiovascular Disease
    Elevated Lp(a)Atherosclerotic Cardiovascular Disease
  • SLN312SLN312 / ANGPTL3 / siRNA / Cardiometabolic
    Dyslipidemia
  • SLN548SLN548 / CFB / siRNA / Complement
  • SLN365SLN365 / GPR146 / siRNA / Cardiometabolic
    Homozygous familial hypercholesterolemia (HoFH)
  • SLN098SLN098 / INHBE / siRNA / Metabolic
    Obesity
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  • AmezalpatTPST-1120 / PPARα antagonist / Small Molecule / Oncology
    Hepatocellular Carcinoma (HCC)CholangiocarcinomaRenal Cell Carcinoma (RCC)
  • TPST-1495TPST-1495 / Dual EP2/EP4 Antagonist / Small Molecule / Oncology / Gastrointestinal
    Familial Adenomatous Polyposis (FAP)Solid Tumors
  • TREX-1 InhibitorTREX-1 / Small Molecule / Oncology
    Solid Tumors
  • TPST-2003TPST-2003 / CD19/BCMA / Autologous CAR-T Cell Therapy / Oncology
    Relapsed/Refractory Multiple Myeloma (rrMM)
  • TPST-3003TPST-3003 / CD19/BCMA / Allogeneic Cell Therapy / Oncology
    Relapsed/Refractory Multiple Myeloma (rrMM)
  • TPST-4003TPST-4003 / CD19/BCMA / In Vivo Cell Therapy / Immunology / Autoimmune
    Systemic Lupus Erythematosus (SLE)Immunology Disorders
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  • TreprostinilTyvaso / Prostacyclin receptor / Small Molecule / Respiratory
    Idiopathic Pulmonary Fibrosis (IPF)
  • RalinepagProstacyclin receptor / Small Molecule / Cardiopulmonary
    Pulmonary Arterial Hypertension (PAH)
  • UKidneyUKidney / Xenotransplantation / Organ Transplantation
    End Stage Renal Disease
  • UThymoKidneyUThymoKidney / Xenotransplantation / Organ Transplantation
    End Stage Renal Disease
  • miroliverELAPmiroliverELAP / Regenerative Medicine / Organ Transplantation
    Acute Liver Failure
  • Centralized Lung Evaluation System (CLES)Ex-vivo Perfusion System / Organ Transplantation
    Lung Transplant
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  • ElebsiranVIR-2218 / HBV / siRNA / Infectious Diseases
    Chronic Hepatitis Delta (CHD)Phase 3ActiveGlobalChronic Hepatitis B (CHB)Phase 2ActiveGlobal
  • TobevibartVIR-3434 / HBV / Monoclonal Antibody / Infectious Diseases
    Chronic Hepatitis Delta (CHD)Phase 3ActiveGlobalChronic Hepatitis B (CHB)Phase 2ActiveGlobal
  • VIR-1388VIR-1388 / HIV / T Cell Vaccine / Infectious Diseases
    HIV PreventionPhase 1ActiveUnited StatesSouth Africa
  • VIR-5500VIR-5500 / PSMA / Masked T-Cell Engager / Oncology
    Solid TumorsPhase 1ActiveGlobal
  • VIR-5525VIR-5525 / EGFR / Masked T-Cell Engager / Oncology
    Solid TumorsPhase 1ActiveGlobal
  • VIR-5818VIR-5818 / HER2 / Masked T-Cell Engager / Oncology
    Solid TumorsPhase 1ActiveGlobal
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  • VK2735 (Subcutaneous)VK2735 / Dual GLP-1/GIP receptor agonist / Peptide / Metabolic Disorders
    Obesity
  • VK2735 (Oral)VK2735 / Dual GLP-1/GIP receptor agonist / Peptide / Metabolic Disorders
    Obesity
  • VK2809VK2809 / Thyroid hormone receptor beta (TRβ) agonist / Small molecule / Metabolic Disorders
    NASHFibrosisNAFLD
  • VK0214VK0214 / Thyroid hormone receptor beta (TRβ) agonist / Small molecule / Rare Diseases
    X-linked adrenoleukodystrophy (X-ALD)
  • Amylin Agonist (DACRAs)Dual amylin and calcitonin receptor agonist (DACRA) / Agonist / Metabolic Disorders
    ObesityMetabolic disorders
  • VK5211VK5211 / Selective androgen receptor modulator (SARM) / Small molecule / Musculoskeletal Disorders
    Hip fracture recoveryMuscle and bone disorders
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  • fasedienolPH94B / peripheral nasal chemosensory receptors / Intranasal pherine / Psychiatry
    Social Anxiety Disorder (SAD)
  • itruvonePH10 / peripheral nasal chemosensory receptors / Intranasal pherine / Psychiatry
    Major Depressive Disorder (MDD)
  • refisolonePH80 / peripheral nasal chemosensory receptors / Intranasal pherine / Women's Health
    Menopausal Vasomotor SymptomsPremenstrual Dysphoric Disorder (PMDD)
  • PH15PH15 / peripheral nasal chemosensory receptors / Intranasal pherine / Neurology/Psychiatry
    Psychomotor and cognitive impairment due to mental fatigue
  • PH284PH284 / peripheral nasal chemosensory receptors / Intranasal pherine / Oncology Supportive Care
    Cancer Cachexia
  • AV-101AV-101 / NMDA receptor glycine site / Small molecule / Neurology/Psychiatry
    Neurological disorders
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  • VilastobartXTX101 / CTLA-4 / Monoclonal Antibody / Oncology
    Advanced solid tumors / MSS mCRCPhase 2ActiveGlobal
  • Efarindodekin alfaXTX301 / IL-12 / Cytokine / Oncology
    Advanced solid tumorsPhase 2ActiveGlobal
  • XTX501XTX501 / PD-1 / IL-2 / Bispecific Antibody / Cytokine / Oncology
    Non-small cell lung cancer (NSCLC) and other solid tumorsPreclinicalActiveGlobal
  • PSMA+STEAP1 Masked T Cell EngagerPSMA / STEAP1 / T Cell Engager / Oncology
    Prostate cancerPreclinicalActiveGlobal
  • CLDN18.2 Masked T Cell EngagerCLDN18.2 / T Cell Engager / Oncology
    Gastric, pancreatic, esophageal, and lung cancersPreclinicalActiveGlobal
  • Undisclosed Masked T Cell EngagerUndisclosed / T Cell Engager / Oncology
    CancerPreclinicalActiveGlobal
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  • BosakitugATI-045 / TSLP / humanized anti-TSLP monoclonal antibody / immuno-inflammatory; respiratory; dermatology
    atopic dermatitisPhase 2ongoing
  • ATI-052ATI-052 / TSLP; IL-4Rα / humanized bispecific antibody / immuno-inflammatory; respiratory; dermatology
    atopic dermatitisPhase 1bongoingasthmaPhase 1bongoingasthmaPhase 2bplanned
  • ATI-2138ATI-2138 / ITK; JAK3 / oral covalent small-molecule inhibitor / T cell-mediated autoimmune diseases; dermatology
    lichen planusPhase 2bplannedatopic dermatitisPhase 2acompleted
  • ATI-9494ATI-9494 / ITK; TXK/RLK / oral covalent small-molecule inhibitor / Th1, Th2 and Th17-driven diseases
    undisclosed inflammatory diseasespreclinicalIND planned
  • LepzacitinibATI-1777 / JAK1; JAK3 / topical soft small-molecule inhibitor / dermatology
    atopic dermatitisPhase 2bseeking partner
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  • TanruprubartANX005 / C1q / classical complement pathway / IV mAb / Autoimmune / neurology
    Guillain-Barré SyndromeMAA filed; FDA discussions ongoingactiveUSEU
  • VonaprumentANX007 / C1q / classical complement pathway / IVT Fab / Ophthalmology
    Geographic AtrophyPhase 3ARCHER II enrollment completed; data expected 2H26
  • ANX1502ANX1502 / Classical complement pathway / Oral small molecule / Autoimmune
    Cold Agglutinin DiseaseProof-of-conceptactive; update upon CAD study completion in 2026
  • ANX005ANX005 / C1q / classical complement pathway / IV mAb / Neurodegeneration
    Huntington’s DiseasePoised for late-stage Phase 2b/3activeAmyotrophic Lateral SclerosisPoised for late-stage Phase 2b/3active
  • ANX009ANX009 / C1q / classical complement pathway / Subcutaneous Fab / Autoimmune
    Lupus NephritisEvaluating options for future development
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  • TransCon hGHSKYTROFA / growth hormone / TransCon prodrug therapy / Endocrinology Rare Diseases
    Turner syndrome (children aged 1–10)Phase 2United StatesMulti-Indication: Turner syndrome, ISS, SHOX Deficiency, SGA (children aged 2–17)Phase 3MultinationalPediatric GHDPhase 3Japan
  • TransCon CNPC-type natriuretic peptide / TransCon prodrug therapy / Endocrinology Rare Diseases
    Achondroplasia (infants aged 0–<2)Pivotal Phase 2MultinationalAchondroplasia (children aged 2–11)Pivotal Phase 2MultinationalAchondroplasia (adolescents aged 12–18)Pivotal Phase 2bMultinational
  • TransCon CNP + TransCon hGHCNP; growth hormone / TransCon prodrug therapy combination / Endocrinology Rare Diseases
    Achondroplasia (children aged 2–11)Phase 2Multinational
  • TransCon PTHYORVIPATH / parathyroid hormone / TransCon prodrug therapy / Endocrinology Rare Diseases
    Adult Hypoparathyroidism (dosing expansion)Phase 3United StatesAdult HypoparathyroidismPhase 3China
  • TransCon IL-2 β/γ (Systemic)IL-2 β/γ / TransCon prodrug therapy / Oncology
    Various tumor typesPhase 2Multinational
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  • FirmonertinibEGFR TKI / oral small molecule / Oncology
    1L NSCLC EGFR exon 20 insertion mutationsPhase 3Monotherapyglobal1L+ NSCLC EGFR PACC mutationsPhase 3MonotherapyglobalAdjuvant EGFR uncommon mutationsPhase 3Monotherapy
  • ARR-217ARR-217 / CDH17 ADC / antibody-drug conjugate / Oncology
    GI tumorsPreclinical
  • ARR-002ARR-002 / antibody-drug conjugate / Oncology
    Solid tumorsDiscovery
  • ARR-421ARR-421 / antibody-drug conjugate / Oncology
    Solid tumorsDiscovery
  • ARR-173ARR-173 / antibody-drug conjugate / Oncology
    Solid tumorsDiscovery
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  • AcoramidisAttruby / TTR (Transthyretin) / Small Molecule / Genetic disease
    Early-Stage Variant Transthyretin Amyloidosis (ATTRv)Phase 3Under investigation
  • BBP-418BBP-418 / FKRP / Small Molecule / Genetic disease; neuromuscular
    Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)Phase 3Investigational; FORTIFY interim analysis met endpoints
  • EncaleretCASR / Small Molecule / Endocrine; genetic disease
    Autosomal Dominant Hypocalcemia Type 1 (ADH1)Phase 3NDA planned/submission stated for H1 2026; pediatric registrational trial initiatedUSEUChronic HypoparathyroidismPhase 2NIH investigator-initiated study; Phase 3 planned in 2026
  • InfigratinibFGFR3 / Small Molecule / Skeletal dysplasia; genetic disease
    AchondroplasiaPhase 3PROPEL 3 registrational study; infant/toddler study enrollingUSEUHypochondroplasiaPhase 2ACCEL 2/3 Phase 2 portion dosed; data expected H2 2026
  • BBP-812BBP-812 / ASPA / Gene Therapy / Genetic disease; neurology
    Canavan DiseaseInvestigational AAV gene therapy
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  • ORLADEYO (berotralstat)ORLADEYO / plasma kallikrein / oral small-molecule inhibitor / Rare diseases
    Hereditary angioedema (HAE) prophylaxisApproved/Commercialmarketed
  • ORLADEYO (berotralstat) in PediatricsORLADEYO / plasma kallikrein / oral small-molecule inhibitor / Rare diseases
    Hereditary angioedema (HAE) prophylaxis, ages 2 to <12Approved/Commercialmarketed
  • NavenibartSTAR-0215 / plasma kallikrein / monoclonal antibody inhibitor / Rare diseases
    Hereditary angioedema (HAE) prophylaxisPhase 3/Pivotalinvestigational
  • BCX17725BCX17725 / KLK5 / protein therapeutic / Rare diseases
    Netherton syndromePhase 1investigational
  • STAR-0310STAR-0310 / OX40 / monoclonal antibody antagonist / Immunology/Dermatology
    Atopic dermatitisPreclinicalinvestigational; strategic alternatives planned
5assets
  • nuzefatide pevedotinBT5528 / EphA2 / Bicycle Drug Conjugate / Oncology
    High EphA2-expressing tumorsPhase I/IIOngoingMetastatic pancreatic ductal adenocarcinomaPhase IIOngoing
  • zelenectide pevedotinBT8009 / Nectin-4 / Bicycle Drug Conjugate / Oncology
    Nectin-4-expressing tumorsPhase I/IIOngoing Duravelo-1Metastatic urothelial cancerPhase IIOngoing Duravelo-2
  • BT7480BT7480 / Nectin-4/CD137 / Bicycle TICA / Oncology
    Advanced solid tumors associated with Nectin-4 expressionPhase I/IIOngoing; seeking partner for future development
  • BT1702BT1702 / MT1-MMP / Bicycle Radioligand / Oncology
    MT1-MMP-expressing tumorsIND-enabling / human imagingCompany-sponsored BRC clinical trial planned in 2027
  • ION286/AZD4063ION286/AZD4063 / TfR1 / TfR1-targeted Bicycle conjugate / Cardiomyopathy
    CardiomyopathyPhase 1Ongoing
5assets
  • BMN 333BMN 333 / C-type natriuretic peptide pathway / Long-acting CNP peptide / Skeletal Conditions
    AchondroplasiaPhase 2/3Registration-enabling study enrolling
  • BMN 351BMN 351 / Dystrophin / Oligonucleotide / Innovation
    Duchenne muscular dystrophyPhase 1/2Clinical data update expected year-end 2026
  • BMN 401BMN 401 / ENPP1 / Subcutaneous enzyme replacement therapy / Enzyme Therapies
    ENPP1 deficiencyPhase 3ENERGY 3 met 1 of 2 co-primary endpoints; next steps under evaluation
  • vosoritideVOXZOGO / CNP receptor/NPR-B pathway / Peptide / Skeletal Conditions
    HypochondroplasiaPhase 3Positive pivotal data; sNDA planned Q3 2026USEUAchondroplasiasNDAFull approval sNDA submitted April 2026USHypochondroplasia under 3 years oldPhase 2Enrollment progressing
  • pegvaliase-pqpzPALYNZIQ / Phenylalanine ammonia lyase / Enzyme therapy / Enzyme Therapies
    Phenylketonuria adolescents 12+RegulatoryFDA approved February 2026; EU approval expected 2026USEU
5assets
  • BDC-4182BDC-4182 / Claudin 18.2 / Boltbody ISAC / Oncology
    Gastric cancerPhase 1Ongoing dose-escalationGastroesophageal cancerPhase 1Ongoing dose-escalation
  • BDC-3042BDC-3042 / Dectin-2 / Agonist monoclonal antibody / Oncology
    Non-small cell lung cancer, melanoma and other solid tumorsPhase 1Dose-escalation complete; seeking partner
  • CEA ISACCEA/CEACAM5 / Boltbody ISAC / Oncology
    Colorectal, non-small cell lung, pancreatic and gastric cancersPreclinicalPre-IND; seeking partner
  • PD-L1 ISACPD-L1 / Boltbody ISAC / Oncology
    Solid tumors resistant to checkpoint inhibitorsPreclinicalPre-IND; seeking partner
  • Caprin-1 ISACCaprin-1 / Boltbody ISAC / Oncology
5assets
  • Lasme-celCD22 / allogeneic CAR-T / Oncology
    relapsed/refractory B-cell acute lymphoblastic leukemiaPhase 2BALLI-01; pivotal Phase 2 first interim analysis expected Q4 2026
  • Eti-celCD20, CD22 / dual allogeneic CAR-T / Oncology
    relapsed/refractory Non-Hodgkin lymphomaPhase 1NatHaLi-01; full Phase 1 dataset expected Q4 2026
  • Cemacabtagene ansegedleucelCema-cel / CD19 / allogeneic CAR-T / Oncology
    large B-cell lymphomaPivotal Phase 2ALPHA3
  • ALLO-316ALLO-316 / CD70 / allogeneic CAR-T / Oncology
    advanced or metastatic clear cell renal cell carcinomaPhase 1TRAVERSE
  • IOV-4001IOV-4001 / PD-1 / TALEN-modified tumor-infiltrating lymphocyte therapy / Oncology
    unresectable or metastatic melanomaIOV-GM1-201stage III or IV NSCLCIOV-GM1-201
5assets
  • CLS-AX (axitinib)CLS-AX / VEGF receptors-1, -2 and -3 / Small molecule tyrosine kinase inhibitor / Ophthalmology
    Wet AMDPhase 3 readyFDA end-of-Phase 2 alignment
  • XIPERE (triamcinolone acetonide injectable suspension)XIPERE; ARCATUS / Corticosteroid injectable suspension / Ophthalmology
    Uveitic macular edemaApproved/commercialUnited StatesAustraliaSingaporeUveitic macular edemaClinical development/commercialization partnerChinaDiabetic macular edemaClinical development/commercialization partnerAsia Pacific ex-Japan
  • Bel-SarViral-like drug conjugate / Ophthalmology/oncology
    Choroidal melanomaPartner clinical development program
  • ABBV-RGX-314ABBV-RGX-314 / anti-VEGF / AAV gene therapy / Ophthalmology
    Diabetic retinopathyPartner clinical development programWet AMDPartner clinical development program
  • AvoralstatPlasma kallikrein / Small molecule plasma kallikrein inhibitor / Ophthalmology
    Diabetic macular edemaPartner clinical development program
5assets
  • bezuclastinibCGT9486 / KIT exon 17/KIT D816V / small molecule tyrosine kinase inhibitor / Oncology; hematology
    NonAdvanced Systemic MastocytosisNDA acceptedFDA review; PDUFA December 30, 2026USGastrointestinal Stromal Tumors after prior imatinibNDA submittedFDA reviewUSAdvanced Systemic Mastocytosisregistration-directed trial completedNDA planned 1H 2026US
  • CGT4255CGT4255 / mutant ErbB2/HER2 / CNS-penetrant small molecule inhibitor / Oncology
    Advanced solid tumors with HER2 mutations or HER2 overexpressionPhase 1dose escalation ongoing
  • CGT6297CGT6297 / PI3Kα / small molecule inhibitor / Oncology
    PI3Kα-mutant cancersPhase 1initiated
  • CGT1815CGT1815 / pan-KRAS(ON) / small molecule inhibitor / Oncology
  • CGT1145CGT1145 / JAK2 V617F / small molecule inhibitor / Hematology
5assets
  • COYA 302COYA 302 / Treg enhancement; T effector and macrophage depletion / Treg-enhancing/T effector and macrophage-depleting biologic combination; low-dose IL-2 + CTLA4-Ig / Neurodegenerative diseases
    Amyotrophic lateral sclerosisPhase 2Fast Track; ALSTARS Phase 2 enrollingUSCanadaFrontotemporal dementiaIND acceptedactiveUSAlzheimer's diseasePreclinical/POCactive
  • COYA 301COYA 301 / IL-2; Treg function and expansion / Proprietary low-dose IL-2 biologic / Neurodegenerative diseases
    Alzheimer's diseaseIND-enablingactive
  • COYA 303COYA 303 / IL-2; GLP-1 receptor / COYA 301 + GLP-1 receptor agonist combination / Inflammatory diseases
    UndisclosedDiscoveryactive
  • COYA 201COYA 201 / Allogeneic Treg-derived exosomes / Undisclosed
    UndisclosedPreclinicalactive
  • COYA 206COYA 206 / Antigen-directed allogeneic Treg-derived exosomes / Undisclosed
    UndisclosedPreclinicalactive
5assets
  • EB613EB613 / PTH 1-34, teriparatide / oral peptide tablet / Osteoporosis
    Postmenopausal women with low BMD and high-risk osteoporosisPhase 3Phase 3 protocol submitted to FDA; registrational program plannedU.S.
  • EB612EB612 / LA-PTH 1-34 / oral long-acting PTH analog peptide tablet / Hypoparathyroidism
    HypoparathyroidismPreclinicalDevelopment ongoing; pre-IND initiatives underway
  • OXMOPK-88006 / GLP-1 & glucagon agonist / oral oxyntomodulin peptide tablet / Obesity / metabolic diseases
    Obesity and metabolic diseasesPreclinicalIND filing expected late H1 2026U.S.
  • GLP-2Long-acting GLP-2 / oral GLP-2 peptide tablet / Short bowel syndrome
    Short bowel syndromePreclinicalProof-of-concept single-dose pharmacokinetic study completed in rodents
  • EB613EB613 / PTH 1-34, teriparatide / oral peptide tablet / Stress fractures
    Stress fracturesInvestigator Sponsored Trial
5assets
  • HydronidoneF351 / TGFβRI/p38γ (anti-fibrotic) / Small molecule / Fibrosis
    CHB-associated liver fibrosisNDA (priority review)NDA submission for conditional approval expected 1H 2026; Breakthrough Therapy designation (NMPA, Mar 2021)ChinaMASH-associated liver fibrosisIND-enablingU.S. IND application anticipated in 2026United States
  • PirfenidoneETUARY / Anti-fibrotic / Small molecule / Fibrosis/Respiratory
    Idiopathic pulmonary fibrosis (IPF)Approved/MarketedApproved in China (2011); commercializedChinaPneumoconiosis (PD)Phase 352-week Phase 3 trial enrollment completed (272 patients, 18 sites)ChinaRadiation-induced lung injury (RILI) with or without immune-related pneumonitisClinicalIn developmentChina
  • F573F573 / Caspase inhibitor / Small molecule / Hepatology
    Acute/acute-on-chronic liver failure (ALF/ACLF)ClinicalIn developmentChina
  • F528F528 / Small molecule / Respiratory
    Chronic obstructive pulmonary disease (COPD)PreclinicalIn developmentChina
  • F230F230 / Small molecule / Cardiopulmonary
    Pulmonary arterial hypertension (PAH)PreclinicalIn developmentChina
5assets
  • CrofelemerMytesi / CFTR and CaCC channels / Botanical Drug / Gastrointestinal
    HIV-Related DiarrheaApprovedMarketedUnited StatesCancer Therapy-related Diarrhea (CTD) / Chemotherapy-induced overactive bowelPhase 3ActiveUnited StatesIrritable Bowel SyndromeIn DevelopmentActiveUnited States
  • Crofelemer Powder for Oral SolutionCFTR and CaCC channels / Botanical Drug / Gastrointestinal / Rare Disease
    Microvillus Inclusion Disease (MVID)Late StageActiveUnited StatesEuropeShort Bowel Syndrome with Intestinal Failure (SBS-IF)Late StageActiveUnited StatesEurope
  • NP-300NP-300 / Unknown / Botanical Drug / Gastrointestinal / Infectious Disease
    Cholera Diarrhea / Infectious DiarrheaIn DevelopmentActiveGlobal
  • GelclairGelclair / Unknown / Bioadherent Oral Gel / Supportive Care / Oncology
    Oral MucositisApprovedMarketedUnited States
  • Crofelemer (Animal Health)Canalevia-CA1 / CFTR and CaCC channels / Botanical Drug / Animal Health
    Chemotherapy-induced diarrhea in dogsApprovedMarketedUnited States
5assets
  • KRRO-121KRRO-121 / Glutamine Synthetase (GS) / GalNAc-conjugated oligonucleotide (RNA Editing) / Metabolic / Liver
    Urea Cycle Disorders (UCD) / Hepatic Encephalopathy (HE)PreclinicalActiveGlobal
  • AATD GalNAc ProgramSERPINA1 mRNA / GalNAc-conjugated oligonucleotide (RNA Editing) / Rare Diseases / Respiratory / Liver
    Alpha-1 Antitrypsin Deficiency (AATD)PreclinicalActiveGlobal
  • AMPKγ1 ProgramAMPKγ1 / GalNAc-conjugated oligonucleotide (RNA Editing) / Metabolic / Liver
    Liver Function / LongevityPreclinicalActiveGlobal
  • ALS ProgramTDP-43 mRNA / Oligonucleotide (RNA Editing) / Neurology
    Amyotrophic Lateral Sclerosis (ALS)PreclinicalActiveGlobal
  • Cardiometabolic ProgramUndisclosed / Oligonucleotide (RNA Editing) / Metabolic / Cardiovascular
    Cardiometabolic DiseasePreclinicalActiveGlobal
5assets
  • ziftomenibKOMZIFTI / Menin / Small Molecule / Oncology
    Acute Myeloid Leukemia (AML) (Combination)Phase 3ActiveGlobalRelapsed or refractory (R/R) NPM1-mutant AMLPre-RegistrationNDA Submitted / Priority ReviewUS
  • rocatinlimabKHK4083 / AMG 451 / OX40 / Monoclonal Antibody / Immunology/Allergy
    Moderate to Severe Atopic DermatitisPhase 3ActiveGlobalUSEUJPPrurigo NodularisPhase 3ActiveGlobalAsthmaPhase 2ActiveGlobal
  • atidarsagene autotemcelOTL-200 / Lenmeldy / Libmeldy / ARSA / Hematopoietic Stem Cell (HSC) Gene Therapy / Rare Diseases
    Early-onset Metachromatic Leukodystrophy (MLD)Pre-RegistrationPreparation underway for clinical trial / Regulatory ReviewJP
  • tivozanibKHK4951 / VEGF Receptor Tyrosine Kinase / Small Molecule / Ophthalmic
    Ophthalmic DiseasesPhase 2/3ActiveGlobal
  • mogamulizumabKW-0761 / POTELIGEO / CCR4 / Monoclonal Antibody / Oncology
    Mycosis fungoides and Sézary syndromePre-RegistrationFiledCanada
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  • sparsentansparsentan / Filspari / ETA/AT1 receptors / Small molecule / Nephrology
    IgA Nephropathy
  • ensifentrineensifentrine / Ohtuvayre / PDE3/PDE4 / Small molecule / Respiratory
    COPD
  • pneumococcal 15-valent conjugate vaccineV114 / Vaxneuvance / Pneumococcal serotypes / Vaccine / Infectious Disease
    Pneumococcal disease
  • carfilzomibcarfilzomib / Kyprolis / Proteasome / Small molecule / Oncology
    Multiple Myeloma
  • berdazimerberdazimer / Zelsuvmi / Nitric oxide / Small molecule / Dermatology
    Molluscum contagiosum
5assets
  • CertepetideLSTA1 / alpha-v integrins / Cyclic Peptide / Oncology
    Advanced Solid TumorsPancreatic Ductal Adenocarcinoma (PDAC)Cholangiocarcinoma (CCA)
  • LSTA12LSTA12 / HONEDRA / CD34+ / Cell Therapy / Cardiovascular
    Critical Limb Ischemia (CLI)
  • LSTA14LSTA14 / OLOGO / CD34+ / Cell Therapy / Cardiovascular
    No-Option Refractory Disabling Angina (NORDA)
  • XOWNAXOWNA / CD34+ / Cell Therapy / Cardiovascular
    Coronary Microvascular Dysfunction (CMD)
  • LSTA201LSTA201 / CD34+ / Cell Therapy / Nephrology
    Diabetic Kidney Disease (DKD)
5assets
  • LP-300LP-300 / PTPN11 / Small molecule / Oncology
    Non-small cell lung cancer (NSCLC) in never-smokers
  • LP-184LP-184 / PTGR1-activated alkylating agent / Small molecule / Oncology
    Solid tumorsCNS cancersGlioblastoma multiforme (GBM)
  • LP-284LP-284 / PKC-beta / Small molecule / Oncology
    Hematologic malignanciesAggressive B-cell lymphomas
  • STAR-001STAR-001 / Unknown / Small molecule / Oncology
    CNS cancersBrain metastases
  • ADC ProgramUnknown / Antibody-drug conjugate (ADC) / Oncology
    Cancer
5assets
  • VDPHL01VDPHL01 / Small molecule / Dermatology
    Androgenetic Alopecia (Pattern Hair Loss)
  • VDAAVDAA / Topical small molecule / Dermatology
    Alopecia Areata
  • VDADVDAD / Dermatology
    Atopic Dermatitis
  • VDMCVDMC / Dermatology
    Molluscum Contagiosum
  • VDMN-21VDMN-21 / Immune stimulatory / Microarray / Dermatology
    Warts
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  • MaralixibatLIVMARLI / IBAT / Small molecule / Rare Liver Diseases
    Alagille syndrome (ALGS)Progressive familial intrahepatic cholestasis (PFIC)Biliary atresia
  • VolixibatSHP626 / IBAT / Small molecule / Rare Liver Diseases
    Primary sclerosing cholangitis (PSC)Primary biliary cholangitis (PBC)
  • MRM-3379MRM-3379 / PDE4D / Small molecule / Neurocognitive Disorders
    Fragile X syndrome
  • Cholic acidCHOLBAM / Bile acid replacement / Small molecule / Rare Liver Diseases
    Bile acid synthesis disordersPeroxisomal disorders (Zellweger spectrum disorders)
  • ChenodiolCTEXLI / Bile acid replacement / Small molecule / Rare Genetic Disorders
    Cerebrotendinous xanthomatosis (CTX)
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  • ALXN1840ALXN1840 / Copper / Small Molecule / Rare Diseases
    Wilson Disease
  • MNPR-101-ZrMNPR-101-Zr / uPAR / Radiopharmaceutical / Oncology
    Advanced Solid Cancers (Imaging)
  • MNPR-101-LuMNPR-101-Lu / uPAR / Radiopharmaceutical / Oncology
    Advanced Solid Cancers
  • MNPR-101-AcMNPR-101-Ac / uPAR / Radiopharmaceutical / Oncology
    Advanced Solid Cancers
  • MNPR-101MNPR-101 / uPAR / Monoclonal Antibody / Oncology
    Solid Tumors
5assets
  • PetosemtamabMCLA-158 / EGFR x LGR5 / Bispecific Antibody / Oncology
    1L PD-L1+ r/m head and neck squamous cell carcinoma (with pembrolizumab)Phase 3OngoingGlobal2/3L r/m head and neck squamous cell carcinomaPhase 3OngoingGlobal1L metastatic colorectal cancer (with standard chemotherapy)Phase 2OngoingGlobal
  • ZenocutuzumabMCLA-128 / BIZENGRI / HER2 x HER3 / Bispecific Antibody / Oncology
    Pancreatic adenocarcinoma (NRG1 fusion-positive)ApprovedApprovedUnited StatesNon-small cell lung cancer (NRG1 fusion-positive)ApprovedApprovedUnited StatesOther non-NRG1+ cancerPhase 2OngoingGlobal
  • MCLA-129MCLA-129 / EGFR x c-MET / Bispecific Antibody / Oncology
    Solid tumorsPhase 1/Phase 2OngoingGlobal2L+ EGFRm non-small cell lung cancer (with chemotherapy)Phase 2OngoingGlobal
  • BesufetamigONO-4685 / PD-1 x CD3 / Bispecific Antibody / Oncology, Immunology
    Relapsed/Refractory T Cell LymphomaPhase 1OngoingGlobalPsoriasisPhase 1OngoingGlobal
  • INCA33890INCA33890 / PD-1 x TGFBr2 / Bispecific Antibody / Oncology
    Select advanced solid tumorsPhase 1OngoingGlobal
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  • navacaprantNMRA-140 / Kappa opioid receptor (KOR) antagonist / Small molecule / Neuropsychiatry
    Major Depressive Disorder
  • NMRA-511NMRA-511 / Vasopressin 1a receptor (V1aR) antagonist / Small molecule / Neuropsychiatry / Neurodegenerative
    Alzheimer's disease agitation
  • NMRA-215NMRA-215 / NLRP3 inhibitor / Small molecule / Endocrinology / Metabolic
    Obesity
  • NMRA-861NMRA-861 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / Neuropsychiatry
    SchizophreniaNeuropsychiatric disorders
  • NMRA-898NMRA-898 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / Neuropsychiatry
    SchizophreniaNeuropsychiatric disorders
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  • triamcinolone acetonideZILRETTA / Small Molecule Formulation / Musculoskeletal Pain
    Shoulder Osteoarthritis (OA)Phase 3Active
  • iovera°iovera° / Device / Neurology/Pain
    SpasticityClinicalActive
  • bupivacaine liposomeEXPAREL / Small Molecule Formulation / Pain Management
    Acute Post-Surgical Pain (Pediatric 0 to < 6)ClinicalActive
  • enekinragene inzadenovecPCRX-201 / IL-1Ra / Gene Therapy / Musculoskeletal Pain
    Osteoarthritis of the Knee (OAK)Phase 2Active
  • PCRX-2002PCRX-2002 / Unknown / Pain Management
    Post-Surgical PainClinicalActive
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  • VAX-31VAX-31 / Streptococcus pneumoniae (31 Serotypes) / Pneumococcal Conjugate Vaccine (PCV) / Infectious Diseases
    Invasive Pneumococcal Disease (IPD) and Pneumonia in AdultsPhase 3OngoingUnited StatesInvasive Pneumococcal Disease (IPD) in InfantsPhase 2OngoingUnited States
  • VAX-24VAX-24 / Streptococcus pneumoniae (24 Serotypes) / Pneumococcal Conjugate Vaccine (PCV) / Infectious Diseases
    Invasive Pneumococcal Disease (IPD) in InfantsPhase 2OngoingUnited States
  • VAX-A1VAX-A1 / Group A Streptococcus / Conjugate Vaccine / Infectious Diseases
    Group A Strep InfectionsPreclinicalOngoing
  • VAX-GIVAX-GI / Shigella / Vaccine / Infectious Diseases
    Shigellosis / Shigella InfectionsPreclinicalOngoing
  • VAX-XLVAX-XL / Streptococcus pneumoniae / Pneumococcal Conjugate Vaccine (PCV) / Infectious Diseases
    Invasive Pneumococcal Disease (IPD)PreclinicalOngoing
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  • PMN310PMN310 / Amyloid-Beta / Monoclonal Antibody / Neurology
    Alzheimer's Disease
  • PMN267PMN267 / TDP-43 / Monoclonal Antibody / Neurology
    Amyotrophic Lateral Sclerosis (ALS)Frontotemporal Dementia (FTD)Limbic-predominant age-related TDP-43 encephalopathy (LATE)
  • PMN442PMN442 / Alpha-Synuclein / Monoclonal Antibody / Neurology
    Parkinson's Disease (PD)Multiple System Atrophy (MSA)Dementia with Lewy Bodies (DLB)
  • PMN311PMN311 / Amyloid-Beta / Vaccine / Neurology
    Alzheimer's Disease
  • PMN440PMN440 / Alpha-Synuclein / Vaccine / Neurology
    Parkinson's Disease
5assets
  • deupirfenidoneLYT-100 / Small Molecule / Respiratory / Fibrotic Diseases
    Idiopathic Pulmonary Fibrosis (IPF)Phase 3ActiveGlobal
  • LYT-200LYT-200 / Biologic / Oncology
    Relapsed/Refractory High-Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)Phase 1ActiveGlobalSolid TumorsPhase 1ActiveGlobal
  • GlyphAlloSPT-300 / Allopregnanolone / Small Molecule / Neuropsychiatry
    Anxiety and Mood DisordersPhase 2ActiveGlobal
  • GlyphAgoSPT-320 / Small Molecule / Neuropsychiatry
    Neurological / Neuropsychiatric IndicationsPhase 1ActiveGlobal
  • xanomeline and trospium chlorideKarXT / Cobenfy / Muscarinic Receptors / Small Molecule / Neuropsychiatry
    SchizophreniaApprovedActiveUS
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  • AMT-130AMT-130 / Huntingtin protein (mHTT) / Gene Therapy (AAV5) / Central Nervous System
    Huntington's DiseasePhase 1/2ActiveGlobal
  • AMT-191AMT-191 / Alpha-galactosidase A (GLA) / Gene Therapy / Rare Diseases
    Fabry DiseasePhase 1/2aActiveGlobal
  • AMT-260AMT-260 / Undisclosed / Gene Therapy / Central Nervous System
    Refractory Mesial Temporal Lobe EpilepsyPhase 1/2aActiveGlobal
  • AMT-162AMT-162 / SOD1 / Gene Therapy / Central Nervous System
    Amyotrophic Lateral Sclerosis (ALS)Preclinical/ClinicalActiveGlobal
  • etranacogene dezaparvovecAMT-061 / HEMGENIX / Factor IX / Gene Therapy (AAV5) / Hematology
    Hemophilia BApprovedMarketedUSGlobal
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  • BrepocitinibJAK1 / TYK2 / Small Molecule / Autoimmune & Inflammatory
    DermatomyositisPhase 3ActiveGlobalNon-infectious UveitisPhase 3ActiveGlobalCutaneous SarcoidosisPhase 2ActiveGlobal
  • IMVT-1402IMVT-1402 / FcRn / Monoclonal Antibody / Autoimmune
    Rheumatoid Arthritis (difficult-to-treat)Phase 2ActiveGlobalGraves' DiseasePhase 2/3ActiveGlobal
  • BatoclimabFcRn / Monoclonal Antibody / Autoimmune
    Thyroid Eye Disease (TED)Phase 3ActiveGlobalMyasthenia Gravis (MG)Phase 3ActiveGlobalChronic Inflammatory Demyelinating Polyneuropathy (CIDP)Phase 2bActiveGlobal
  • MosliciguatRVT-304 / sGC / Small Molecule / Pulmonary
    Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)Phase 2ActiveGlobal
  • NamilumabGM-CSF / Monoclonal Antibody / Autoimmune & Inflammatory
    SarcoidosisPhase 2ActiveGlobal
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  • RopidoxuridineIPdR / Radiation Sensitizer / Small Molecule / Oncology
    GlioblastomaPhase 2ActiveUnited StatesSarcomasPhase 1/Phase 2ActiveUnited States
  • SP-2-225SP-2-225 / HDAC6 / Small Molecule / Oncology
    Breast Cancer / Solid TumorsPreclinicalActiveUnited States
  • SP-1-303SP-1-303 / Class I HDAC / Small Molecule / Oncology
    ER+ Breast CancerPreclinicalActiveUnited States
  • SP-1-161SP-1-161 / HDAC / Small Molecule / Oncology
    Solid TumorsDiscoveryActiveUnited States
  • PSMA-B ligandPSMA-B / PSMA / Radiopharmaceutical / Oncology
    Metastatic Prostate CancerPreclinicalActiveUnited States
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  • MR-146MR-146 / AAV Gene Therapy / Ophthalmology
    Neurotrophic keratopathy (NK)
  • Low Dose Estrogen Weekly PatchEstrogen / Progestin receptors / Transdermal Patch / Women's Health
    Contraception
  • PitolisantHistamine H3 receptor / Small molecule / Neurology
    Obstructive Sleep Apnea Syndrome
  • SelatogrelP2Y12 receptor / Small molecule / Cardiovascular
    Acute Myocardial Infarction
  • CenerimodS1P1 receptor / Small molecule / Immunology
    Systemic lupus erythematosus (SLE)
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  • WVE-006WVE-006 / AAT transcript (Pi*ZZ mutation) / RNA editing oligonucleotide (AIMer) / Genetic/Respiratory/Liver Diseases
    Alpha-1 antitrypsin deficiency (AATD)
  • WVE-007WVE-007 / INHBE mRNA / siRNA / Metabolic Diseases
    Obesity
  • WVE-008WVE-008 / PNPLA3 I148M / RNA editing oligonucleotide / Liver Diseases
    PNPLA3-related liver disease
  • WVE-N531WVE-N531 / DMD Exon 53 / Exon skipping oligonucleotide / Neuromuscular Diseases
    Duchenne muscular dystrophy (DMD)
  • WVE-003WVE-003 / Mutant huntingtin (mHTT) mRNA / Allele-selective silencing oligonucleotide / Neurological Diseases
    Huntington's disease (HD)
4assets
  • ABCL635ABCL635 / NK3R / antibody medicine / Endocrinology / Women's Health
    Vasomotor Symptoms (VMS) associated with menopausePhase 2active
  • ABCL575ABCL575 / OX40L / monoclonal antibody / Immunology & Inflammation
    Atopic Dermatitis (AD)Phase 1active
  • ABCL688ABCL688 / Undisclosed GPCR / Ion Channel / antibody medicine / Autoimmunity
    Undisclosed autoimmunity indicationIND-Enablingactive
  • ABCL386ABCL386 / Undisclosed / antibody medicine / Oncology
    Undisclosed oncology indicationIND-Enablingactive
4assets
  • prademagene zamikeracelpz-cel / ZEVASKYN / COL7A1 / autologous cell-based gene therapy / Dermatology / rare disease
    recessive dystrophic epidermolysis bullosa (RDEB)Approvedapproved
  • PSMA SIR-TABO-701 / PSMA / engineered autologous T-cell therapy / Oncology
    Advanced prostate cancerPreclinicalpreclinical
  • UX111UX111 / SGSH / in vivo AAV9 gene therapy / Rare disease / CNS
    Sanfilippo Syndrome Type A (MPS IIIA)Phase 3clinical
  • TSHA-102TSHA-102 / MECP2 / intrathecal AAV9 gene transfer therapy / Rare disease / CNS
    Rett SyndromePhase 1/2clinical
4assets
  • MitapivatPYRUKYND®; AQVESME™ / PK / PK activator / Rare diseases; hematology
    Adult PK deficiencyApprovedAdult thalassemiaApprovedU.S.KSAUAEAdult sickle cell diseaseRegulatory submissionU.S.
  • TebapivatPK / PK activator / Rare diseases; hematology
    Lower-risk myelodysplastic syndromesClinical developmentSickle cell diseaseClinical development
  • AG-181AG-181 / PAH / PAH stabilizer / Rare diseases; metabolic disorders
    PhenylketonuriaClinical development
  • AG-236AG-236 / TMPRSS6 / siRNA / Rare diseases; hematology
    Polycythemia veraClinical development
4assets
  • PraliciguatsGC / soluble guanylate cyclase stimulator / Kidney disease
    Focal segmental glomerulosclerosisPhase 2first patient enrolled Dec 2025
  • Ebribafusp alfaAKB-097 / C3d/factor H / anti-C3d-factor H fusion protein; complement inhibitor / Rare kidney diseases
    IgA nephropathyPhase 2 plannedbasket trial planned 2H 2026Lupus nephritisPhase 2 plannedbasket trial planned 2H 2026C3 glomerulopathyPhase 2 plannedbasket trial planned 2H 2026
  • AKB-9090AKB-9090 / HIF-PH / HIF-PH inhibitor / Kidney disease; acute care
    Cardiac surgery-associated acute kidney injuryPhase 1first participants dosed Apr 2026Acute respiratory distress syndromePreclinical
  • AKB-10108AKB-10108 / HIF-PH / HIF-PH inhibitor / Other disease areas
    Retinopathy of prematurityPreclinical
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  • EnvudeucitinibESK-001 / TYK2 / oral small molecule TYK2 inhibitor / Immunology
    Plaque psoriasisPhase 3ONWARD3 LTE ongoing; NDA planned 2H 2026GlobalJapanSystemic lupus erythematosusPhase 2LUMUS ongoing; topline data expected 2026Global
  • A-005A-005 / TYK2 / CNS-penetrant allosteric TYK2 inhibitor / Neuroinflammation
    Multiple sclerosisPhase 1 completed; Phase 2 plannedPhase 2 initiation planned H1 2026
  • LonigutamabIGF-1R / subcutaneous humanized IgG1 monoclonal antibody / Endocrinology/Immunology
    Thyroid eye diseaseDevelopment program evaluation ongoing
  • IRF5/Additional TargetsIRF5, additional targets
    Undisclosed
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  • AvexitideGLP-1 receptor / GLP-1 receptor antagonist / Endocrine/metabolic diseases
    Post-bariatric hypoglycemia (PBH)Phase 3LUCIDITY completed enrollment; topline data expected Q3 2026USCongenital hyperinsulinism (HI)Clinical development next steps under discussionUS
  • AMX0035AMX0035 / ER stress and mitochondrial dysfunction / Oral fixed-dose combination of sodium phenylbutyrate and taurursodiol / Neurodegenerative/rare diseases
    Wolfram syndromePhase 2HELIOS positive Week 48 data; pivotal Phase 3 planned pending FDA alignmentUSEU
  • AMX0114AMX0114 / Calpain-2 (CAPN2) / Antisense oligonucleotide (ASO) / Neurodegenerative diseases
    Amyotrophic lateral sclerosis (ALS)Phase 1LUMINA Cohort 2 fully enrolled; biomarker data expected H1 2026US
  • AMX0318AMX0318 / GLP-1 receptor / Long-acting GLP-1 receptor antagonist / Endocrine/metabolic diseases
    PBH and other rare diseasesIND-enablingIND-enabling studies underway; IND targeted for 2027
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  • ZumilokibartAPG777 / IL-13 / subcutaneous half-life extended monoclonal antibody / immunology and inflammation
    Atopic dermatitisPhase 2in-progress; Part B enrollment completedAsthmaPhase 1complete; positive Phase 1b dataEosinophilic esophagitisPhase 1in-progress
  • APG279APG279 / IL-13 + OX40L / coformulation of zumilokibart (APG777) + APG990 monoclonal antibodies / immunology and inflammation
    Atopic dermatitisPhase 1in-progress; Phase 1b PoC fully enrolled
  • APG273APG273 / IL-13 + TSLP / combination of zumilokibart (APG777) + APG333 monoclonal antibodies / respiratory immunology and inflammation
    Asthma / COPDPreclinicalin-progress
  • APG808APG808 / IL-4Rα / antibody / respiratory immunology and inflammation
    AsthmaPhase 1bpositive interim results announced May 2025
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  • BPL-003BPL-003 / Intranasal mebufotenin benzoate / Mental health
    Treatment-resistant depressionPhase 2In development
  • VLS-01VLS-01 / 5-HT1/2/6/7 receptor agonist / Oral transmucosal DMT buccal film / Mental health
    Treatment-resistant depressionPhase 2In development
  • EMP-01EMP-01 / Oral R-MDMA / Mental health
    Social anxiety disorderPhase 2Positive topline results announced
  • Novel 5-HT2A Receptor Agonists5-HT2A receptor / Small-molecule neuroplastogens / Mental health
    Opioid use disorderDiscoveryIn developmentTreatment-resistant depressionDiscoveryIn development
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  • DeramiocelCAP-1002 / allogeneic CDCs / Neuromuscular disease
    Duchenne muscular dystrophy (DMD)Phase 3in progressBecker muscular dystrophy (BMD)Preclinicalin progress
  • StealthX™STX-S / Exosome-based vaccine / Infectious disease
    SARS-CoV-2Phase 1in progress
  • StealthX™Therapeutic Exosomes
    EvaluatingPreclinicalin progress
  • CDC-ExosomesExosome Platform / Neuromuscular disease
    Duchenne muscular dystrophyPreclinicalin progress
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  • CLN-978CLN-978 / CD19xCD3 / bispecific T cell engager / Immunology
    OUTRACE: Rheumatoid arthritisPhase 1Initial data in RA in Q2 2026 and repeat dosing data in Q3 2026EuropeOUTRACE: Systemic lupus erythematosusPhase 1bInitial data in SLE in Q2 2026GlobalOUTRACE: Sjögren’s diseasePhase 1Initial data in Sjögren’s disease in Q4 2026Global
  • VelinotamigBCMAxCD3 / bispecific T cell engager / Immunology
    Autoimmune diseasesIND-EnablingGenrix Bio initiated a Phase 1 study in China in Q4 2025; initial data expected in Q4 2026; clinical trial status not yet recruitingChina
  • ZipalertinibEGFR ex20ins / tyrosine kinase inhibitor (TKI) / Oncology
    REZILIENT1: Non-small cell lung cancerPhase 2U.S. FDA accepted NDA for relapsed EGFR ex20ins NSCLC; PDUFA target action date February 27, 2027United StatesREZILIENT2: Non-small cell lung cancerPhase 2Parallel cohort study ongoingREZILIENT3: Non-small cell lung cancerPhase 3Phase 3 1L study fully enrolled; Taiho expects topline results by end of 2026Global
  • CLN-049CLN-049 / FLT3xCD3 / bispecific T cell engager / Oncology
    Acute myeloid leukemia and myelodysplastic syndromePhase 1Dose escalation update in H2 2026; dose expansion cohorts to initiate in Q2 2026 and complete enrollment in Q4 2026Measurable residual disease in acute myeloid leukemiaPhase 1Phase 1 study ongoing
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  • COM701COM701 / PVRIG / Fc-reduced anti-PVRIG antibody / Oncology
    Relapsed platinum-sensitive ovarian cancer; maintenance therapyAdaptive platform trialongoing
  • COM701 + COM902 + pembrolizumabPVRIG; TIGIT; PD-1 / Antibody combination / Oncology
    Platinum-resistant ovarian cancerProof of concept
  • rilvegostomigPD-1/TIGIT / Fc-reduced anti-PD-1/TIGIT bispecific antibody / Oncology
    NSCLCPhase 3; Phase 1/2ongoingGastrointestinal cancerPhase 3; Phase 1/2ongoingEndometrial cancerPhase 3ongoing
  • GS-0321GS-0321 / IL18BP / Anti-IL18BP antibody / Oncology
    Solid tumorsPhase 1ongoing
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  • CNM-Au8®CNM-Au8 / mitochondrial function / NAD pathway; neuroprotection and remyelination / oral gold nanocrystal suspension / Neurology
    Amyotrophic Lateral Sclerosis (ALS)Global Phase 3 plannedPlanned; EAPs ongoing; regulatory agency engagement ongoingUSEUGlobalMultiple Sclerosis (MS)Phase 3 plannedPlanned; EAP ongoing; Repair-MS target engagement and safety confirmedUSGlobalParkinson’s Disease (PD)In development; REPAIR-PD trial listed
  • CNM-ZnAgCNM-ZnAg / zinc-silver ionic solution / Infectious disease
    Antiviral and antimicrobial usesDemonstrated antiviral and antimicrobial properties
  • CNM-AgZn17CNM-AgZn17 / topical gel polymer suspension of silver and zinc ions / Infectious disease / wound healing
    Infectious diseasesIn developmentWound healingIn development
  • CNM-PtAu7CNM-PtAu7 / gold-platinum nanotherapeutic / Oncology
    CancerIn vitroInitial research
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  • tovecimigCTX-009 / DLL4 x VEGF-A / bispecific antibody / Oncology
    Biliary tract cancer (2L)Phase 2/3Met primary endpoint; PFS/OS expected late Q1 2026Colorectal cancer (monotherapy 3L/4L)Phase 2CompletedDLL4+ tumors basket studyAdditional indications planned H1 2026
  • CTX-471CTX-471 / CD137 / agonistic antibody / Oncology
    NCAM (CD56) basket studyTrial initiation expected Q1 2026Post-checkpoint basket studyCompleted
  • CTX-8371CTX-8371 / PD-1 x PD-L1 / bispecific antibody / Oncology
    Solid tumorsPhase 1Expansion cohorts initiated; Phase 1 data expected H1 2026
  • CTX-10726CTX-10726 / PD-1 x VEGF-A / bispecific antibody / Oncology
    Solid tumorsIND-enabling preclinicalPhase 1 initiation expected Q1 2026
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  • PIPE-791LPA1R / small molecule LPA1R antagonist / inflammation; immunology; neuroscience
    Idiopathic pulmonary fibrosisPhase 2activeProgressive MSPhase 1deferredChronic painPhase 1bactive
  • CTX-343LPA1R / peripherally restricted small molecule LPA1R antagonist / peripheral indications
    PeripheralPreclinicaldeferred
  • PIPE-307JNJ-5120 / M1R / small molecule M1R antagonist / neuroscience
    Relapsing-remitting MSPhase 2licensedMajor depressive disorderPhase 2licensed
  • CalpainCalpain / calpain inhibitor
    UndisclosedPreclinicalactive
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  • AficamtenMYQORZO / cardiac myosin inhibitor / small molecule / Cardiovascular
    oHCMApproved & CommercializedApproved in U.S., China, & EUU.S.ChinaEUoHCM (vs. metoprolol)Phase 3MAPLE-HCMPediatric oHCMPhase 2CEDAR-HCM
  • Ulacamtencardiac myosin inhibitor / small molecule / Cardiovascular
    HFpEFPhase 2AMBER-HFpEF
  • Omecamtiv Mecarbilcardiac myosin activator / small molecule / Cardiovascular
    HFrEFPhase 3COMET-HF
  • CK-089CK-089 / fast skeletal muscle troponin activator / small molecule / Neuromuscular
    Muscular Dystrophy, OtherPhase 1
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  • DT-216P2DT-216P2 / FXN GAA repeat expansion / frataxin expression / GeneTAC small molecule / Genetic disease / neurology
    Friedreich ataxiaPhase 1/2RESTORE-FA multiple-dose trial opened/ongoing; evaluates IV and subcutaneous administrationex-U.S.
  • DT-168DT-168 / TCF4 CTG repeat expansion / GeneTAC small molecule eye drop / Ophthalmology / genetic disease
    Fuchs endothelial corneal dystrophyPhase 2Biomarker trial opened/ongoing in FECD patients scheduled for corneal transplant surgery
  • DT-818DT-818 / DMPK CTG repeat expansion / GeneTAC small molecule / Neuromuscular / genetic disease
    Myotonic dystrophy type 1Phase 1 plannedPatient dosing in Phase 1 MAD trial anticipated in H1 2026Australia
  • Huntington's disease GeneTAC programHTT CAG repeat expansion / mutant huntingtin expression / GeneTAC small molecule / Neurology / genetic disease
    Huntington's diseasePreclinicalProgram disclosed; candidate not named
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  • Halneuron® NaV1.7 InjectionHAL / Halneuron® / NaV1.7 / subcutaneous small-molecule/peptide analgesic injection / Pain / neuropathy
    Chemotherapy-induced neuropathic pain (CINP)Phase 2bOngoing; FDA Fast Track designationU.S.General cancer painPhase 2aCompleteAcute surgical painPreclinical
  • SP16 Intravenous AdministrationSP16 IV / LRP1 agonist / IV peptide therapeutic / Neuropathy / cancer-related pain
    Chemotherapy-induced peripheral neuropathy (CIPN)Phase 1bNCI funded / forthcoming
  • IMC-1IMC-1 / Herpesvirus suppression / COX-2 inhibition / Fixed-dose combination of anti-herpesvirus medication and COX-2 inhibitor / Pain / fatigue illness
    FibromyalgiaPhase 2aPreviously demonstrated efficacy; disclosed as a novel therapeutic under antiviral combinations
  • IMC-2IMC-2 / Herpesvirus suppression / COX-2 inhibition / Fixed-dose combination of anti-herpesvirus medication and COX-2 inhibitor / Pain / fatigue illness
    Long COVIDOpen-label investigator-initiated study completed/reported; disclosed as a novel therapeutic under antiviral combinations
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  • SevasemtenMyosin ATPase / Oral fast skeletal myosin inhibitor / Muscular dystrophy
    Becker muscular dystrophyPivotal / Phase 3Duchenne muscular dystrophyPhase 2
  • EDG-7500EDG-7500 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / Cardiovascular
    Hypertrophic cardiomyopathyPhase 2
  • EDG-15400EDG-15400 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / Cardiovascular
    HFpEFPhase 1
  • EDG-003EDG-003 / Undisclosed target / Cardiometabolic
    CardiometabolicPreclinical
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  • 4D-1504D-150 / VEGF family members / Intravitreal AAV genetic medicine / Retina
    Wet AMDPivotal / Phase 34FRONT-1 and 4FRONT-2 Phase 3; PRISM Phase 1/2 ongoingDiabetic macular edemaPivotal readySPECTRA clinical trial Part 1
  • 4D-7104D-710 / CFTR / Aerosol-delivered AAV genetic medicine / Pulmonology
    Cystic fibrosis lung disease; not modulator-amenable / ineligible or discontinued CFTR modulator therapyPhase 2AEROW Phase 1/2 study; pipeline lists Phase 2
  • 4D-1754D-175 / Complement factor H / complement pathway / Intravitreal AAV genetic medicine / Retina
    Geographic atrophySeeking partnerships to advance
  • 4D-7254D-725 / Aerosol-delivered AAV genetic medicine / Pulmonology
    Alpha-1 antitrypsin lung diseasePreclinicalSeeking partnerships to advance
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  • MRT-6160MRT-6160 / VAV1 / Oral molecular glue degrader / Immunology & Inflammation
    Immune-mediated diseasesClinical
  • MRT-8102MRT-8102 / NEK7 / Oral molecular glue degrader / Immunology & Inflammation
    IL-1β/NLRP3-driven inflammatory diseasesPhase 1
  • MRT-2359MRT-2359 / GSPT1 / Oral molecular glue degrader / Oncology
    Castration-resistant prostate cancerPhase 1/2Ongoing
  • Cyclin E1 degrader programCCNE1 / CDK2 / Molecular glue degrader / Oncology
    CCNE1-amplified tumorsPreclinicalLead optimizationER+ breast cancerPreclinicalLead optimization
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  • HCW9302HCW9302 / IL-2Rα / IL-2 fusion protein / Autoimmune
    Alopecia areataPhase 1OngoingUS
  • HCW11-006HCW11-006 / Multiple cytokines (IL-15/IL-15Rα, IL-7, IL-21, IL-2) / Multi-functional fusion immunotherapeutic / Oncology
    CancerPhase 1Planned by licenseeChina
  • HCW11-018bHCW11-018b / Tissue Factor/CD3 / Tetra-valent bispecific T-cell engager (Big BiTE) / Oncology
    Solid tumorsPreclinicalIND-enablingGlobal
  • HCW11-040HCW11-040 / PD-1 (pembrolizumab-based) + TGF-β / Tetra-valent immune checkpoint inhibitor fusion / Oncology
    Solid tumorsPreclinicalIND-enablingGlobal
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  • nogapendekin alfa inbakiceptN-803 / ANKTIVA® / IL-15 receptor / Cytokine fusion protein / Interleukin-15 superagonist / Oncology, Infectious Diseases
    BCG Unresponsive NMIBC Carcinoma In-Situ & Papillary (QUILT-3.032)ApprovedMarketedUSBCG Naïve NMIBC (QUILT-2.005)Phase 2/Phase 3ActiveUSNon-Small Cell Lung Cancer (ResQ201A)Phase 3ActiveGlobal
  • PD-L1 t-haNKPD-L1 / Cell Therapy / Engineered NK cells / Oncology
    Advanced Solid TumorsPhase 2ActiveUS
  • AldoxAlbumin-bound doxorubicin complex / Oncology
    Advanced Solid TumorsPhase 2ActiveUS
  • Recombinant BCGVaccine / Immunotherapy / Oncology
    Bladder CancerExpanded AccessActiveUS
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  • anzu-cel (IMA203)IMA203 / anzutresgene autoleucel / PRAME / Autologous TCR-T cell therapy / Solid Tumors
    2L+ cutaneous melanomaPhase 3ongoingUSEU5Uveal melanomaPhase 1bongoingUSEU5PRAME+ solid tumorsPhase 1ongoingUSEU5
  • IMA203CD8 (GEN2)IMA203CD8 / PRAME / Autologous TCR-T cell therapy (next-gen with CD8) / Solid Tumors
    PRAME+ advanced solid tumorsPhase 1dose escalation ongoingUSEU5Ovarian and endometrial cancersPhase 1ongoingUSEU5
  • IMA402IMA402 / PRAME / Half-life extended TCR bispecific (TCER) / Solid Tumors
    Recurrent/refractory solid tumors (melanoma, gynecologic, sqNSCLC)Phase 1/2Phase 1b expansion ongoingUSEU
  • IMA401IMA401 / MAGEA4/8 / TCR bispecific (TCER) / Solid Tumors
    Recurrent/refractory solid tumors (HNSCC, sqNSCLC, melanoma)Phase 1dose escalation completedUSEU
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  • INB-100INB-100 / DeltEx Allo allogeneic γδ T cell therapy / Oncology
    Acute leukemia post-HSCTPhase 1Expansion cohort enrollingUS
  • INB-200INB-200 / DeltEx DRI autologous genetically modified γδ T cell therapy / Oncology
    Newly diagnosed glioblastomaPhase 1Treatment completed; long-term follow-upUS
  • INB-400INB-400 / DeltEx DRI autologous and allogeneic γδ T cell therapy / Oncology
    Newly diagnosed glioblastomaPhase 2Enrollment suspended; treatment completed; data collection ongoingUS
  • INB-619INB-619 / CD19 / γδ T cell engager (TCE) / Oncology and autoimmune diseases
    CD19+ B-cell malignanciesPreclinicalIND-enabling studiesB-cell mediated autoimmune diseasesPreclinicalIND-enabling studies
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  • agenT-797agenT-797 / Allogeneic native iNKT cell therapy / Oncology and Immunology
    Critical pulmonary immune failure (severe hypoxemic respiratory failure/pneumonia/ARDS)Phase 2randomized adaptive placebo-controlled ongoingUSSolid tumor cancers (checkpoint-refractory, incl. gastric/esophageal/GEJ adenocarcinoma)Phase 2investigator-sponsored ongoing (NCT06251973)USGraft-versus-host disease (GvHD prevention)expanding development
  • MiNK-215MiNK-215 / FAP / IL-15 armored FAP-CAR-iNKT cell therapy / Oncology
    Solid tumors (NSCLC, CRC liver metastases)PreclinicalIND-enabling studies underway
  • MiNK-413MiNK-413 / BCMA / IL-15 armored BCMA-CAR-iNKT cell therapy / Oncology
    Multiple myelomaPreclinical
  • PRAME TCR-iNKTPRAME / TCR-engineered iNKT cell therapy / Oncology
    Pediatric cancers/solid tumorsPreclinicalIND-enabling
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  • bitopertinGlyT1 / Small Molecule / Hematology
    Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP)Phase 3OngoingGlobalDiamond-Blackfan AnemiaPhase 2OngoingUS
  • DISC-0974DISC-0974 / Hemojuvelin (HJV) / Monoclonal Antibody / Hematology
    Anemia of Myelofibrosis (MF)Phase 2OngoingGlobalAnemia of Chronic Kidney Disease (CKD)Phase 1/Phase 2OngoingGlobalAnemia of Inflammatory Bowel Disease (IBD)Phase 2PlannedGlobal
  • DISC-3405DISC-3405 / TMPRSS6 (Matriptase-2) / Monoclonal Antibody / Hematology
    Polycythemia Vera (PV)Phase 2OngoingGlobalSickle Cell Disease (SCD)Phase 1bOngoingGlobal
  • DISC-0998DISC-0998 / Hemojuvelin (HJV) / Monoclonal Antibody / Hematology
    Inflammatory-disease-associated anemiaPreclinicalOngoingGlobal
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  • SelinexorKPT-330 / XPOVIO / XPO1 (Exportin 1) / Small Molecule / Oncology
    Multiple MyelomaMarketedActiveUSGlobalEndometrial CancerPhase 3ActiveUSGlobalMyelofibrosisPhase 3ActiveUSGlobal
  • EltanexorKPT-8602 / XPO1 (Exportin 1) / Small Molecule / Oncology
    Myelodysplastic NeoplasmsPhase 2PausedUSColorectal CancerPhase 2PausedUSProstate CancerPhase 2PausedUS
  • KPT-9274KPT-9274 / PAK4 / NAMPT / Small Molecule / Oncology
    Solid Tumors & LymphomaPhase 1PausedUS
  • VerdinexorKPT-335 / XPO1 (Exportin 1) / Small Molecule / Veterinary Oncology / Infectious Diseases
    Canine LymphomaMarketedActiveUS
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  • IFx-2.0IFx-Hu2.0 / Innate Immune System / Personalized Cancer Vaccine / Innate Immune Agonist / Oncology
    Advanced or Metastatic Merkel Cell Carcinoma (1st line, adjunctive to pembrolizumab)Phase 3OngoingUnited StatesMerkel Cell Carcinoma of Unknown Primary (MCCUP)Phase 1/Phase 2OngoingUnited States
  • TBS-2025KVA12123 / VISTA / Monoclonal Antibody / Oncology
    Advanced Solid Tumor CancerPhase 1OngoingUnited StatesmutNPM1 Acute Myeloid Leukemia (AML)Phase 1/Phase 2PlannedUnited States
  • Bi-functional ADCsMyeloid-derived suppressor cells (MDSCs) / Antibody-Drug Conjugate (ADC) / Oncology
    Solid TumorsPreclinicalOngoingUnited States
  • REM-001Unknown / Photodynamic Therapy / Oncology
    Cutaneous Metastatic Breast Cancer (CMBC)Phase 3OngoingUnited States
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  • PAS-004PAS-004 / MEK 1/2 / Macrocyclic Small Molecule / Oncology / Rare Diseases
    Neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN)MAPK pathway-driven advanced solid tumors
  • PAS-003PAS-003 / α5β1 Integrin / Monoclonal Antibody / Neurology
    Amyotrophic Lateral Sclerosis (ALS)
  • PAS-001PAS-001 / C4A / Small Molecule / Psychiatry
    Schizophrenia
  • PAS-002PAS-002 / Neurology
    Multiple Sclerosis (MS)
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  • ZiftomenibKO-539 / KOMZIFTI / Menin-KMT2A/MLL interaction / Small molecule / Hematologic Malignancies
    R/R NPM1-mutated AMLNewly Diagnosed AMLFLT3-mutated AML
  • DarlifarnibKO-2806 / Farnesyl transferase / Small molecule / Oncology
    Clear cell renal cell carcinoma (ccRCC)Solid tumors
  • TipifarnibFarnesyl transferase / Small molecule / Oncology
    HRAS-mutant Head and Neck Squamous Cell Carcinoma (HNSCC)PIK3CA-dependent HNSCC
  • KO-7246KO-7246 / Menin / Small molecule / Cardiometabolic
    DiabetesCardiometabolic disease
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  • SAT-3247SAT-3247 / AAK1 / Small molecule / Degenerative muscle diseases
    Duchenne Muscular Dystrophy (DMD)Facioscapulohumeral Muscular Dystrophy (FSHD)
  • SAT-3153SAT-3153 / Notch polarity kinase / Small molecule / Degenerative muscle diseases
    Duchenne Muscular Dystrophy (DMD)
  • BertilimumabiCo-008 / Eotaxin-1 (CCL11) / Monoclonal antibody / Immunology
    Bullous Pemphigoid
  • iCo-019iCo-019 / Fungal cell membrane / Small molecule (Oral Amphotericin B) / Infectious Diseases
    Fungal infections
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  • GivastomigCLDN18.2 x 4-1BB / Bispecific Antibody / Oncology
    Gastric CancerGastrointestinal Malignancies
  • RagistomigPD-L1 x 4-1BB / Bispecific Antibody / Oncology
    Solid Tumors
  • UliledlimabCD73 / Monoclonal Antibody / Oncology
    Cancer
  • VIS-101VEGF-A x ANG-2 / Bispecific Antibody / Ophthalmology
    Wet Age-Related Macular Degeneration (AMD)Diabetic Macular Edema (DME)
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  • NEO100NEO-100 / Unknown / Small Molecule / Oncology, Neurology
    Malignant GliomasGrade 4 AstrocytomaParkinson's Disease
  • NEO212NEO-212 / DNA / Small Molecule Conjugate / Oncology
    Malignant Gliomas
  • NEO214NEO-214 / DR5, PDE4 / Small Molecule Conjugate / Oncology
    Glioblastoma (GBM)Multiple Myeloma
  • NEO400NEO-400 / Unknown / Small Molecule Conjugate / Dermatology, Oncology
    UV-Induced Skin DamageTopical Oncology
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  • TaletrectinibIBTROZI / ROS1 / Small Molecule / Oncology
    Advanced ROS1+ NSCLC
  • SafusidenibmIDH1 / Small Molecule / Oncology
    Diffuse IDH1-mutant gliomaAstrocytomaOligodendroglioma
  • NUV-1511NUV-1511 / Undisclosed / Drug-Drug Conjugate (DDC) / Oncology
    Advanced solid tumors
  • NUV-868NUV-868 / BET (BD2-selective) / Small Molecule / Oncology
    Advanced solid tumors
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  • NarsoplimabOMS721 / Yartemlea / MASP-2 / Monoclonal antibody / Hematology / Complement-Mediated Diseases
    Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)
  • ZaltenibartOMS906 / MASP-3 / Monoclonal antibody / Hematology / Nephrology
    Paroxysmal nocturnal hemoglobinuria (PNH)C3 glomerulopathy
  • OMS1029OMS1029 / MASP-2 / Biologic / Complement-Mediated Diseases
  • OMS527OMS527 / PDE7 / Small molecule / Addiction
    Cocaine use disorder
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  • UlixacaltamidePRAX-944 / T-type calcium channel / Small molecule / Neurology
    Essential Tremor
  • RelutriginePRAX-562 / Persistent sodium current (NaV) / Small molecule / Neurology
    Developmental and Epileptic Encephalopathies (DEEs)SCN2A-DEESCN8A-DEE
  • VormatriginePRAX-628 / Sodium channels (NaV) / Small molecule / Neurology
    Focal Onset EpilepsyEpilepsy
  • ElsunersenSCN2A mRNA / Antisense oligonucleotide (ASO) / Neurology
    SCN2A Developmental and Epileptic Encephalopathy
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  • ZovegalisibRLY-2608 / Mutant PI3Kα / Small molecule / Oncology & Genetic Disease
    HR+/HER2- metastatic breast cancer (PI3Kα-mutated)Phase 3ActiveGlobalPIK3CA-driven vascular malformations/anomaliesPhase 1ActiveGlobal
  • LirafugratinibRLY-4008 / FGFR2 / Small molecule / Oncology
    FGFR2-altered solid tumorsClinicalOut-licensedGlobal
  • RLY-8161RLY-8161 / NRAS / Small molecule / Oncology
    NRAS-driven solid tumorsPreclinicalActiveGlobal
  • Fabry disease programUndisclosed / Non-inhibitory chaperone / Genetic Disease
    Fabry diseasePreclinicalActiveGlobal
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  • SCY-770PXL-770 / AMP-activated protein kinase (AMPK) / Small Molecule / Rare Diseases / Nephrology
    Autosomal Dominant Polycystic Kidney Disease (ADPKD)Phase 2ActiveGlobal
  • SCY-247SCY-247 / 1,3-beta-glucan synthase / Small Molecule / Infectious Diseases
    Treatment and Prevention of Invasive Fungal InfectionsPhase 1ActiveUnited States
  • Fungerp analogs1,3-beta-glucan synthase / Small Molecule / Infectious Diseases
    Treatment of resistant fungiPreclinicalActiveGlobal
  • IbrexafungerpSCY-078 / BREXAFEMME / 1,3-beta-glucan synthase / Small Molecule / Infectious Diseases
    Vulvovaginal candidiasis (VVC) and Recurrent VVCApprovedActiveUnited StatesInvasive Candidiasis (IC)Phase 3ActiveGlobal
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  • SEP-479SEP-479 / PTH1R / Small Molecule / Endocrinology
    HypoparathyroidismPhase 1Active
  • SEP-631SEP-631 / MRGPRX2 / Small Molecule / Immunology and Inflammation
    Chronic spontaneous urticaria (CSU)Phase 1ActiveOther mast cell diseasesPhase 1Active
  • TSHR ProgramTSHR / Small Molecule / Endocrinology
    Graves' diseasePreclinicalActiveThyroid eye diseasePreclinicalActive
  • Novo Nordisk Partnered ProgramsGLP-1R, GIPR, GCGR, Undisclosed / Small Molecule / Metabolic Diseases
    Metabolic diseasesDiscoveryActive
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  • SPC-15SPC-15 / 5-HT4 receptor agonist / Small Molecule / Psychiatry
    PTSD and stress-induced anxiety disordersPreclinicalActiveGlobal
  • SP-26SP-26 / NMDA receptor antagonist / Implant / Neurology
    Fibromyalgia and chronic painPreclinicalActiveGlobal
  • SPC-14SPC-14 / Unknown / Small Molecule / Neurology
    Alzheimer's diseasePreclinicalActiveGlobal
  • SPU-16SPU-16 / Unknown / Peptide / Neurology
    Multiple sclerosisPreclinicalActiveGlobal
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  • patidegibSGT-610 / Hedgehog signaling pathway (SMO) / Small molecule / Dermatology
    Gorlin syndrome (prevention of new BCC lesions)High-frequency basal cell carcinoma (BCC)
  • erlotinibSGT-210 / EGFR / Small molecule / Dermatology
    Undisclosed indications
  • tretinoin and benzoyl peroxide cream, 0.1%/3%TWYNEO / Retinoid receptors / Bacterial proteins / Small molecule / Dermatology
    Acne vulgaris
  • benzoyl peroxide cream, 5%EPSOLAY / Bacterial proteins / Small molecule / Dermatology
    Inflammatory lesions of rosacea
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  • TSC-100TSC-100 / HA-1 / TCR-T Cell Therapy / Hematologic Malignancies
    Acute Myeloid Leukemia (AML) (Post-HCT Relapse Prevention)Phase 1ActiveUnited StatesAcute Lymphoblastic Leukemia (ALL) (Post-HCT Relapse Prevention)Phase 1ActiveUnited StatesMyelodysplastic Syndromes (MDS) (Post-HCT Relapse Prevention)Phase 1ActiveUnited States
  • TSC-101TSC-101 / HA-2 / TCR-T Cell Therapy / Hematologic Malignancies
    Acute Myeloid Leukemia (AML) (Post-HCT Relapse Prevention)Phase 1ActiveUnited StatesAcute Lymphoblastic Leukemia (ALL) (Post-HCT Relapse Prevention)Phase 1ActiveUnited StatesMyelodysplastic Syndromes (MDS) (Post-HCT Relapse Prevention)Phase 1ActiveUnited States
  • ImmunoBank TCR-T CandidatesT-Plex / Multiple Targets (e.g., PRAME, HPV16, MAGE-A1) / Multiplexed TCR-T Cell Therapy / Oncology
    Solid TumorsPhase 1ActiveUnited States
  • Novel TargetsUnidentified Autoantigens / Targeted Therapeutics / Autoimmune Disorders
    Crohn's DiseaseDiscoveryActiveUnited States
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  • AzetukalnerXEN1101 / Kv7.2/Kv7.3 potassium channel opener / Small Molecule / Neurology and Psychiatry
    Focal Onset Seizures (FOS)Phase 3OngoingGlobalPrimary Generalized Tonic-Clonic Seizures (PGTCS)Phase 3OngoingGlobalMajor Depressive Disorder (MDD)Phase 3OngoingGlobal
  • XEN1120XEN1120 / Kv7 potassium channel opener / Small Molecule / Pain
    PainPhase 1OngoingUnited States
  • XEN1701XEN1701 / NaV1.7 sodium channel modulator / Small Molecule / Pain
    PainPhase 1OngoingUnited States
  • NBI-921355NBI-921355 / NaV1.2 / NaV1.6 sodium channel inhibitor / Small Molecule / Neurology
    EpilepsyPhase 1OngoingGlobal
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  • SabirnetugACU193 / toxic soluble amyloid beta oligomers (AβOs) / humanized monoclonal antibody; intravenous formulation / Neurology
    Early Alzheimer's diseasePhase 2ongoing ALTITUDE-ADGlobal
  • SabirnetugACU193 / toxic soluble amyloid beta oligomers (AβOs) / humanized monoclonal antibody; subcutaneous formulation / Neurology
    Early Alzheimer's diseasePhase 1clinical development
  • Enhanced Brain Delivery (EBD™)TfR brain transporter + AβO mAb / blood-brain-barrier-penetrating antibody therapy / Neurology
    Early Alzheimer's diseasePreclinicalpreclinicalPre-clinical Alzheimer's diseasePreclinicalpreclinical
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  • Sodium oxybate extended-release oral suspensiononce-nightly extended-release oral suspension / Sleep disorders
    Idiopathic hypersomniaPhase 3Evaluating in REVITALYZ
  • Olanzapine and samidorphanLYBALVI / oral small molecule combination / Psychiatry
    Schizophrenia and bipolar I disorder (pediatric)Phase 3Evaluating in ENLIGHTEN-Youth
  • AlixorextonALKS 2680 / orexin 2 receptor / oral small molecule agonist / Sleep disorders
    Narcolepsy type 1Phase 3Brilliance Studies programNarcolepsy type 2Phase 3Brilliance Studies programIdiopathic hypersomniaPhase 2Vibrance-3
3assets
  • APR-1051APR-1051 / WEE1 kinase / Oral small-molecule WEE1 inhibitor / Oncology
    CCNE1, CCNE2, FBXW7, PPP2R1A advanced solid tumorsPhase 1Monotherapy; potential safety/efficacy clinical data 2Q 2026; complete dose escalation 2Q 2027HPV+ oropharyngeal squamous cell carcinoma; cervical, vaginal or vulvar squamous cell carcinomaPhase 1MonotherapyKRAS G12/13 & TP53 colorectal cancerPhase 1Monotherapy
  • ATRN-119ATRN-119 / Mosipasertib / ATR / Oral macrocyclic small-molecule ATR inhibitor / Oncology
    ATRi synthetic lethal gene alterations in advanced solid tumors (ovarian, CRC, lung)Phase 1/2aMonotherapy; RP2D determinedHematological cancers (AML, MDS)Phase 1/2aMonotherapy; RP2D determined
  • APR-1602APR-1602 / DYRK1 / Oral small-molecule DYRK1 inhibitor / Oncology
    Solid tumors and hematological cancersIND-enablingReady to enter IND-enabling studies; milestone 4Q 2026
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  • AnaphylmAQST-109 / Anaphylm / epinephrine prodrug / dibutepinephrine sublingual film / Allergy/immunology
    Type 1 allergic reactions, including anaphylaxisNDA resubmission plannedclinical/regulatoryUS
  • AQST-108AQST-108 / epinephrine prodrug / epinephrine topical gel / Dermatology
    Multiple potential indicationsPhase 1clinical
  • LibervantLibervant / GABA-A receptor positive allosteric modulator / diazepam buccal film / Neurology
    Acute repetitive seizures / cluster seizuresRegulatory/approvedpipeline-listedUS
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  • Topical Roflumilast CreamARQ-151 / ZORYVE / PDE4 / small molecule topical cream / Dermatology
    Plaque psoriasis 0.3% (2-5 yr)sNDA submittedPDUFA June 29, 2026USAtopic dermatitis 0.05% (3-24 mo)Phase 2planned sNDA submission Q2 2026US
  • Topical Roflumilast FoamARQ-154 / ZORYVE / PDE4 / small molecule topical foam / Dermatology
    Vitiligo 0.3%Phase 2acurrently enrolling; advancement decision Q4 2026Hidradenitis suppurativa 0.3%Phase 2acurrently enrolling; advancement decision Q1 2027
  • ARQ-234ARQ-234 / CD200R / fusion protein biologic / Dermatology
    Atopic dermatitisPhase 1a/1benrolling
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  • AlloNKAB-101 / AlloNK® / CD20 combinations / Allogeneic off-the-shelf non-genetically modified cryopreserved NK cell therapy / Autoimmune disease; oncology
    Refractory rheumatoid arthritisPhase 2aongoing; lead indication; Fast TrackUSSjögren’s diseasePhase 2aongoingUSIdiopathic inflammatory myopathiesPhase 2aongoingUS
  • CAR-NKAB-205 / CD5 / Allogeneic anti-CD5 CAR-NK cell therapy expressing soluble IL-15 / Oncology
    Hematologic malignanciesPreclinicalcollaborator-funded
  • CAR-NKAB-201 / HER2 / Allogeneic anti-HER2 CAR-NK cell therapy expressing soluble IL-15 / Oncology
    Solid tumorsPreclinicalcollaborator-funded; US orphan drug designationUS
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  • EfzofitimodNRP2 modulator / tRNA synthetase-derived biologic immunomodulator / Interstitial lung disease
    Pulmonary sarcoidosisPhase 3SSc-ILDPhase 2Other ILD (CTD-ILD; CHP)Phase 1
  • ATYR0101ATYR0101 / LTBP1 modulator / Fibrosis
    FibrosisPreclinical
  • ATYR0750ATYR0750 / FGFR4 modulator / Liver disorders
    Liver disordersPreclinical
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  • ATR-12ATR-12 / LEKTI replacement / SPINK5 / Topical live biotherapeutic; engineered S. epidermidis expressing rhLEKTI-D6 / Dermatology
    Netherton syndromePhase 1bclinicalUS
  • ATR-04ATR-04 / IL-36g reduction; S. aureus inhibition / Topical live biotherapeutic; auxotrophic engineered S. epidermidis strain SE484 / Dermatology / oncology supportive care
    EGFR inhibitor-associated rashPhase 1/2clinicalUS
  • ATR-01ATR-01 / Filaggrin / FLG / Topical protein candidate; FLG5-6 with cell-penetrating peptide / Dermatology
    Ichthyosis vulgarisPreclinicalpreclinical
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  • BGE - 102BGE-102 / NLRP3 / CNS-penetrant NLRP3 inhibitor / Metabolic diseases
    CV riskPhase 1Diabetic macular edemaPhase 1
  • APJ OralAPJ / APJ agonist / Metabolic diseases
    ObesityLead optimization
  • APJ ParenteralAPJ / APJ agonist / Metabolic diseases
    ObesityLead optimization
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  • VTP-1000VTP-1000 / gluten antigens; mTOR inhibitor co-delivery / antigen-specific immunotherapy; SNAP Tolerance Platform / Autoimmunity
    Celiac diseasePreclinicalPhase 1 SAD data expected Q4 2025; MAD/challenge data in 2026Worldwide rights
  • VTP-300VTP-300 / HBV antigens / HBV immunotherapeutic; ChAdOx/MVA viral vectors / Infectious diseases
    Chronic hepatitis BPhase 2Pursuing partnership opportunities for advancementWorldwide rights
  • VTP-800/850VTP-800/850 / prostate cancer-associated antigens including PSA, PAP, STEAP1 and 5T4 / prostate cancer immunotherapeutic; ChAdOx/MVA viral vectors / Cancer
    Prostate cancerPhase 1Pursuing partnership opportunities for advancementWorldwide rights
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  • LOQTORZI (toripalimab-tpzi)LOQTORZI / PD-1 / Anti-PD-1 monoclonal antibody / Oncology
    1L Nasopharyngeal Carcinoma (combo with chemotherapy)ApprovedApprovedUnited States2L+ Nasopharyngeal Carcinoma (monotherapy)ApprovedApprovedUnited States
  • TagmokitugCHS-114 / CCR8 / Anti-CCR8 monoclonal antibody / Oncology
    4L+ Colorectal Cancer (combo with toripalimab)Phase 22L HNSCC (combo with toripalimab)Phase 12L Gastric Cancer/GEJ/EAC (combo with toripalimab)Phase 1
  • CasdozokitugIL-27 / IL-27 antagonist monoclonal antibody / Oncology
    1L Hepatocellular Carcinoma (combo with toripalimab + bevacizumab)Phase 2
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  • CTx-1301CTx-1301 / PTR multi-core modified-release dexmethylphenidate tablet / CNS
    ADHDNDA submittedSubmitted July 31, 2025US
  • CTx-1302CTx-1302 / PTR multi-core modified-release dextroamphetamine tablet / CNS
    ADHDPreclinical/formulationInitial formulation study plannedUS
  • CTx-2103CTx-2103 / 5-HT1A partial agonist / PTR multi-core modified-release buspirone tablet / CNS
    AnxietyPre-INDClinical trials planned 4Q 2026US
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  • Iopofosine I 131CLR 131 / tumor cells via phospholipid drug conjugate delivery / PDC radiotherapeutic; iodine-131 beta emitter / Oncology
    Relapsed/refractory Waldenström macroglobulinemiaPhase 2 pivotalcompletedRelapsed/refractory multiple myelomaPhase 2bongoingCNS lymphomaPhase 2bongoing
  • CLR 121125CLR 125 / tumor cell nucleus via phospholipid ether platform / PLE radioconjugate; iodine-125 Auger emitter / Oncology
    Relapsed/refractory triple-negative breast cancerPhase 1bongoing
  • CLR 121225CLR 225 / tumor cells via phospholipid ether platform / PLE radioconjugate; actinium-225 alpha emitter / Oncology
    Pancreatic cancerPhase 1planned
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  • Budoprutug IVCD19 / monoclonal antibody / immune-mediated diseases
    Primary Membranous NephropathyPhase 2Immune ThrombocytopeniaPhase 1Systemic Lupus ErythematosusPhase 1
  • Budoprutug SCCD19 / monoclonal antibody / immune-mediated diseases
    Autoimmune DiseasePhase 1healthy volunteer trial
  • CLYM116CLYM116 / APRIL / monoclonal antibody / immune-mediated diseases
    IgA NephropathyPhase 1healthy volunteer trial
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  • cleminorextonORX750 / OX2R / oral orexin receptor 2 agonist / Sleep-wake disorders
    Narcolepsy Type 1 (NT1)Phase 2aactiveNarcolepsy Type 2 (NT2)Phase 2aactiveIdiopathic Hypersomnia (IH)Phase 2aactive
  • ORX142ORX142 / OX2R / oral orexin receptor 2 agonist / Neurology/neurodegenerative disorders
    Neurological & neurodegenerative disordersPhase 1active
  • ORX489ORX489 / OX2R / oral orexin receptor 2 agonist / Neuropsychiatry
    Neuropsychiatric disordersPreclinicalactive
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  • CTIM-76CTIM-76 / CLDN6 x CD3 / TCE bispecific antibody / Oncology
    recurrent ovarian cancer and other advanced solid tumorsPhase 1ongoingplatinum-resistant ovarian cancerPhase 1FDA Fast TrackUS
  • CT-95CT-95 / MSLN x CD3 / TCE bispecific antibody / Oncology
    advanced solid tumorsPhase 1ongoing
  • CT-202CT-202 / Nectin-4 x CD3 / TCE bispecific antibody / Oncology
    Nectin-4-positive solid tumorsPhase 1planned; HREC approved and TGA CTN acknowledgedAustralia
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  • SoquelitinibCPI-818 / ITK / Oral small molecule ITK inhibitor / Immune diseases; oncology
    Relapsed/refractory peripheral T-cell lymphoma (PTCL)Registration Phase 3EnrollingUnited StatesModerate-to-severe atopic dermatitisPhase 2Initiated/enrollingUnited StatesModerate-to-severe atopic dermatitisPhase 1b/2Enrolling; partner-ledChina
  • CiforadenantCPI-444 / Adenosine A2A receptor / Oral small molecule checkpoint inhibitor / Oncology
    Cancer indicationsClinical-stageGreater China
  • MupadolimabCPI-006 / CD73 / Anti-CD73 monoclonal antibody / Oncology
    Non-small cell lung cancer (NSCLC)Phase 1/1bPartner-led evaluationChina
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  • varsetatug masetecanCX-2051 / Varseta-M / EpCAM / Probody ADC; Topo-1 payload / Oncology
    3L+ metastatic colorectal cancerPhase 1active2L/3L mCRC; Varseta-M + bevacizumabtrials anticipated 2H 20262L mCRC; Varseta-M + bevacizumab + chemotherapytrials anticipated 2H 2026
  • CX-801CX-801 / IFN-alpha 2b / Probody cytokine / Oncology
    Advanced/metastatic melanomaPhase 1active
  • CX-908CX-908 / P-Cadherin x CD3 / Probody T-cell engager / Oncology
    Solid tumorsPreclinicalactive
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  • HLP0035-HT2A and other serotonin receptors / Deuterated novel serotonergic agonist; oral / Mental health
    Adjunctive treatment of Major Depressive Disorder (MDD)Phase 3Dosing underway; FDA Breakthrough Therapy Designation
  • HLP004Serotonin receptors / Deuterated novel serotonergic agonist; intramuscular / Mental health
    Generalized Anxiety Disorder (GAD)Phase 2Enrollment complete
  • HLP005CYB005 / Phenethylamines and tryptamines / CNS disorders
    CNS DisordersPreclinical
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  • TovorafenibDAY101 / OJEMDA / RAF / MAPK pathway / Oral small-molecule type II RAF inhibitor / Oncology
    BRAF-altered relapsed pLGGApprovedOJEMDA received FDA accelerated approval; FIREFLY-1 pivotal Phase 2 follow-up ongoing/disclosedU.S.Frontline RAF-altered pLGGPhase 3 / RegistrationalFIREFLY-2 pivotal Phase 3
  • Emiltatug ledadotinEmi-Le / B7-H4 / Antibody-drug conjugate (ADC) / Oncology
    Adenoid cystic carcinoma (ACC), adult and pediatric solid tumorsPhase 1B7-H4-targeted ADC; Phase 1 data anticipated mid-2026 per company investor materials
  • DAY301MTX-13 / CB-002 / PTK7 / Antibody-drug conjugate (ADC) / Oncology
    Adult and pediatric solid tumorsPhase 1Phase 1a data anticipated 2H 2026 per company investor materials
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  • EB06 Anti-CXCL10 Monoclonal antibodyEB06 / CXCL10 / Monoclonal antibody / Dermatological diseases
    VitiligoPhase 2Health Canada CTA approved; IND in processCanadaUnited States
  • EB01 (daniluromer)EB01 / sPLA2 / Topical sPLA2 inhibitor / Dermatological diseases
    Allergic contact dermatitisPhase 2b completedTopline results available
  • ParidiprubartEB05 / TLR4 / Monoclonal antibody / Respiratory diseases
    Acute respiratory distress syndromePhase 3Phase 3 results reported; selected for U.S. government-funded Phase 2 platform studyUnited StatesCanada
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  • ERAS-0015ERAS-0015 / RAS / Small molecule molecular glue / Oncology
    RAS-mutant solid tumorsPhase 1AURORAS-1; in clinicWorldwide
  • ERAS-4001ERAS-4001 / KRAS / Small molecule / Oncology
    KRAS-mutant solid tumorsPhase 1BOREALIS-1; in clinicWorldwide
  • ERAS-12ERAS-12 / EGFR D2/D3 / Large molecule; bispecific antibody / Oncology
    EGFR and RAS/MAPK altered tumorsWorldwide
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  • DMR-001DMR-001 / mutant calreticulin (mutCALR) / Fc-null, half-life extended anti-mutCALR monoclonal antibody; subcutaneous biologic / Hematology/oncology
    mutCALR-driven essential thrombocythemiaPreclinical / IND-enablingIND or CTA submission planned mid-2026; clinical proof-of-concept datasets expected beginning mid-2027USex-USmutCALR-driven myelofibrosisPreclinical / IND-enablingIND or CTA submission planned mid-2026; clinical proof-of-concept datasets expected beginning mid-2027USex-US
  • DMR-002DMR-002 / mutant calreticulin (mutCALR) / mutCALR-targeted mechanism; undisclosed / Hematology/oncology
    mutCALR-driven myeloproliferative neoplasmsPreclinical / IND-enablingIND or CTA submission planned 2H 2026USex-US
  • DMR-003DMR-003 / mutant calreticulin (mutCALR) x CD3 / bispecific antibody; T-cell engager / Hematology/oncology
    mutCALR-driven myeloproliferative neoplasmsPreclinical / IND-enablingIND or CTA submission planned 2027USex-US
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  • quaratusugene ozeplasmid (REQORSA)REQORSA / TUSC2 / non-viral gene therapy / Oncology
    EGFR-mutant NSCLC (osimertinib-resistant)Phase 1/2Phase 2a expansion enrollingUnited Statesextensive-stage SCLC (maintenance with atezolizumab)Phase 1/2expansion enrollingUnited States
  • GPX-002GPX-002 / PDX1 and MafA / AAV gene therapy / Diabetes
    Type 1 DiabetesPreclinicalType 2 DiabetesPreclinical
  • NPRL2 gene therapyNPRL2 / non-viral gene therapy / Oncology
    KRAS/STK11 mutant anti-PD1-resistant NSCLCPreclinical
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  • HLP0035-HT2A (and other serotonin receptors) / Deuterated NSA (oral) / Mental Health
    Adjunctive treatment of major depressive disorder (MDD)Phase 3Dosing underway (PARADIGM program); FDA Breakthrough Therapy DesignationUnited StatesEuropeAustralia
  • HLP004Serotonin receptors / Deuterated NSA (intramuscular) / Mental Health
    Generalized anxiety disorder (GAD)Phase 2Positive topline results reportedUnited States
  • HLP005NSA (phenethylamines and tryptamines) / Mental Health
    CNS disordersPreclinical
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  • IFx-2.0plasmid DNA (innate immune agonist) / Oncology
    Advanced or metastatic Merkel cell carcinoma (first-line, adjunctive to pembrolizumab)Phase 3OngoingUnited StatesMetastatic Merkel cell carcinoma of unknown primary (MCCUP, first-line)Phase 1b/2aOngoingUnited States
  • TBS-2025VISTA / monoclonal antibody / Oncology
    NPM1-mutated relapsed/refractory AML (with menin inhibitor)Phase 1b/2PlannedUnited States
  • DOR bi-specific ADCDelta opioid receptor (DOR) on MDSCs/TAMs / antibody-drug conjugate (ADC) / Oncology
    Acute myeloid leukemia (AML)PreclinicalLead selection
3assets
  • Vidofludimus calciumIMU-838 / DHODH inhibitor and Nurr1 activator / Oral small molecule / Multiple sclerosis
    Relapsing multiple sclerosisPhase 3Ongoing (ENSURE-1/2 fully enrolled; top-line data expected end 2026)GlobalProgressive multiple sclerosisPhase 2Completed (CALLIPER positive; PPMS Phase 3 initiation expected 2026)North AmericaEurope
  • IMU-856IMU-856 / SIRT6 modulator / Oral small molecule / Gastrointestinal diseases
    Celiac diseasePhase 1bCompleted (positive proof-of-concept)Global
  • IMU-381IMU-381 / Nurr1 / Oral small molecule / Autoimmune diseases
    Neurologic, gastrointestinal and other autoimmune diseasesPreclinicalPreclinical
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  • tarcocimab tedromerZenkuda / VEGF / Antibody Biopolymer Conjugate (ABC) / Ophthalmology
    Wet Age-Related Macular Degeneration (wet AMD)Diabetic RetinopathyRetinal Vein Occlusion (RVO)
  • KSI-501KSI-501 / IL-6 / VEGF / Bispecific Antibody Biopolymer Conjugate (ABC) / Ophthalmology
    Wet Age-Related Macular Degeneration (wet AMD)Diabetic Macular Edema (DME)
  • KSI-101KSI-101 / IL-6 / VEGF / Bispecific Protein / Ophthalmology
    Macular Edema Secondary to Inflammation (MESI)
3assets
  • mivocabtagene autoleucelKYV-101 / CD19 / Autologous CAR T-cell therapy / Autoimmune Diseases
    Stiff Person Syndrome (SPS)Phase 2Completed (Preparing BLA)USGeneralized Myasthenia Gravis (gMG)Phase 3OngoingUSLupus Nephritis (LN)Phase 1/2OngoingUSGermany
  • KYV-102KYV-102 / CD19 / Autologous CAR T-cell therapy / Autoimmune Diseases
    Autoimmune DiseasesPhase 1IND ActiveUS
  • KYV-201KYV-201 / CD19 / Allogeneic CAR T-cell therapy / Autoimmune Diseases
    Autoimmune DiseasesPreclinicalActiveUS
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  • SotagliflozinINPEFA / ZYNQUISTA / SGLT1 / SGLT2 / Small Molecule / Cardiometabolic
    Heart FailureApprovedactiveUnited StatesHypertrophic Cardiomyopathy (HCM)Phase 3activeGlobalType 1 Diabetes (T1D)RegistrationactiveUnited States
  • PilavapadinLX9211 / AAK1 / Small Molecule / Pain / Neurology
    Diabetic Peripheral Neuropathic Pain (DPNP)Phase 2bactiveUnited StatesPostherpetic Neuralgia (PHN)Phase 2inactiveUnited States
  • LX9851LX9851 / ACSL5 / Small Molecule / Metabolism
    Obesity / Weight ManagementPhase 1activeGlobal
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  • PH-762PH-762 / PD-1 / INTASYL (sdRNA) / Oncology
    Cutaneous Squamous Cell CarcinomaMelanomaMerkel Cell Carcinoma
  • PH-894PH-894 / BRD4 / INTASYL (sdRNA) / Oncology / Infectious Disease
    Advanced MelanomaMerkel Cell CarcinomaHepatocellular Carcinoma
  • PH-804PH-804 / TIGIT / INTASYL (sdRNA) / Oncology
    Cancer
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  • D-PLEX100D-PLEX100 / Bacterial Ribosome / Small Molecule Formulation / Infectious Diseases / Surgical Site Care
    Prevention of surgical site infections (SSIs) following abdominal colorectal surgeryPhase 3OngoingUnited StatesEuropeIsrael
  • PP03APP03A / GLP-1 Receptor / Peptide Formulation / Metabolic Disease
    Obesity and Type 2 DiabetesPreclinicalOngoing
  • PP04APP04A / OncoPLEX / STING / Small Molecule Formulation / Oncology
    Solid TumorsPreclinicalOngoing
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  • QRX003QRX003 / Serine protease inhibitor / Small molecule / Dermatology
    Netherton SyndromePhase 3OngoingUnited StatesEuropeMiddle EastPeeling Skin SyndromePhase 2OngoingNew Zealand
  • Topical rapamycinQRX009 / mTOR / Small molecule / Dermatology
    Microcystic Lymphatic MalformationsPreclinicalOngoingGlobalVenous MalformationsPreclinicalOngoingGlobalAngiofibromaPreclinicalOngoingGlobal
  • QRX008QRX008 / VLA-4 / VCAM-1 inhibitor / Small molecule / Dermatology
    SclerodermaPreclinicalOngoingGlobal
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  • RAP-219RAP-219 / TARPγ8 AMPAR / Small molecule / Neurology/Psychiatry
    Focal onset seizuresPrimary generalized tonic-clonic seizuresBipolar mania
  • α6β4 nAChR Agonistα6β4 nAChR / Small molecule / Neurology
    Chronic painMigraine
  • α9α10 nAChR Modulatorα9α10 nAChR / Small molecule / Hearing/Vestibular
    Hearing disordersVestibular disorders
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  • PC111PC111 / sFasL (soluble Fas Ligand) / Monoclonal Antibody / Immunology / Dermatology
    PemphigusPreclinicalActiveGlobalStevens-Johnson syndrome / toxic epidermal necrolysis (SJS/TEN)PreclinicalActiveGlobal
  • Intradermal IL-17A/F NanoAbIL-17A / IL-17F / VHH Antibody Fragment (NanoAb) / Immunology / Dermatology
    Mild-to-moderate plaque psoriasisPreclinicalActiveGlobal
  • Systemic Bi-Specific IL-17 NanoAbIL-17A / IL-17F and secondary proprietary target / Bi-specific VHH Antibody Fragment (NanoAb) / Immunology / Dermatology
    Moderate-to-severe plaque psoriasisPreclinicalActiveGlobalPsoriatic arthritisPreclinicalActiveGlobal
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  • DenifanstatASC40 / FASN / Small molecule / Metabolic disease, Dermatology, Oncology
    MASH (F2/F3)Phase 3ActiveModerate to severe acnePhase 3ActiveRecurrent glioblastoma (GBM)Phase 3Active
  • TVB-3567FASN / Small molecule / Dermatology, Oncology
    AcnePhase 1ActiveSolid tumorsPreclinicalActive
  • FASNi TopicalFASN / Small molecule / Dermatology
    AcnePreclinicalActive
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  • TX45TX-000045 / RXFP1 / Fc-relaxin fusion protein / Cardiopulmonary
    PH-HFpEF (Group 2 Pulmonary Hypertension)PH-HFrEF (Group 2 Pulmonary Hypertension)PH-ILD (Group 3 Pulmonary Hypertension)
  • TX2100TX002100 / GPCR / Biotherapeutic / GPCR Antagonist / Hematology / Rare Diseases
    Hereditary Hemorrhagic Telangiectasia (HHT)
  • Fibrosis ProgramGPCR / Bispecific GPCR Antagonist / Fibrosis
    Fibrosis
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  • ublituximabTG-1101 / BRIUMVI / CD20 / Monoclonal Antibody / Autoimmune Diseases
    Relapsing Multiple Sclerosis (RMS)
  • ublituximab subcutaneousCD20 / Monoclonal Antibody / Autoimmune Diseases
    Relapsing Multiple Sclerosis (RMS)
  • azercabtagene zapreleucelazer-cel / CD19 / Allogeneic CAR-T Cell Therapy / Autoimmune Diseases
    Progressive Multiple Sclerosis (PMS)Autoimmune Diseases
3assets
  • Intranasal ForalumabTZLS-401 / CD3 epsilon / Monoclonal Antibody / Neurology/Immunology
    Non-Active Secondary Progressive Multiple Sclerosis (SPMS)Multiple System Atrophy (MSA)Mild Alzheimer's Disease
  • Anti IL-6R mAbTZLS-501 / IL-6R / Monoclonal Antibody / Immunology
  • MilciclibTZLS-201 / CDKs / Small Molecule / Oncology
    NSCLC with pan KRAS-positive mutations
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  • TN-201TN-201 / MYBPC3 / AAV-based gene therapy / Cardiovascular
    MYBPC3-associated Hypertrophic Cardiomyopathy (HCM)
  • TN-401TN-401 / PKP2 / AAV9-based gene therapy / Cardiovascular
    PKP2-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
  • TN-301TN-301 / HDAC6 / Small Molecule / Cardiometabolic / Neuromuscular
    Heart Failure with preserved Ejection Fraction (HFpEF)Duchenne Muscular Dystrophy (DMD)
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  • dabogratinibTYRA-300 / FGFR3 / Small molecule / Oncology / Skeletal Dysplasia
    Metastatic Urothelial Cancer (mUC)Non-Muscle Invasive Bladder Cancer (IR NMIBC)Achondroplasia
  • TYRA-200TYRA-200 / FGFR1/2/3 / Small molecule / Oncology
    Intrahepatic CholangiocarcinomaAdvanced Solid Tumors
  • TYRA-430TYRA-430 / FGFR4/3 / Small molecule / Oncology
    Hepatocellular Carcinoma (HCC)Advanced Solid Tumors
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  • AtaciceptVT-001 / BAFF and APRIL / Recombinant Fusion Protein / Immunology / Nephrology
    IgA Nephropathy (IgAN)BLA SubmittedPriority Review (PDUFA July 2026)USGlobalLupus NephritisPhase 2OngoingGlobalFocal Segmental Glomerulosclerosis (FSGS)Phase 2OngoingGlobal
  • MAU868MAU868 / BK Virus / Monoclonal Antibody / Infectious Disease / Transplantation
    BK Viremia in Kidney Transplant RecipientsPhase 2Evaluating for future developmentGlobal
  • VT-109VT-109 / BAFF and APRIL / Fusion Protein / Immunology
    B Cell Mediated DiseasesPreclinicalActiveGlobal
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  • Avutometinib and DefactinibVS-6766 / VS-6063 / AVMAPKI FAKZYNJA CO-PACK / RAF/MEK and FAK / Small Molecule / Oncology
    KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC)ApprovedActiveUnited StatesRecurrent low-grade serous ovarian cancer (LGSOC)Phase 3ActiveGlobalMetastatic Pancreatic Cancer (PDAC)Phase 2ActiveGlobal
  • AvutometinibVS-6766 / RAF/MEK / Small Molecule / Oncology
    BRAF, KRAS and NRAS-Mutant Solid TumorsPhase 1ActiveGlobal
  • VS-7375GFH375 / KRAS G12D / Small Molecule / Oncology
    KRAS G12D-Mutated Solid TumorsPhase 1/Phase 2ActiveUnited StatesChina
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  • tibulizumabZB-106 / IL-17A and BAFF / Bispecific dual-antagonist antibody / Autoimmune and Inflammatory Diseases
    Hidradenitis suppurativaSystemic sclerosis
  • crebankitugZB-168 / IL-7Rα / Monoclonal antibody / Autoimmune and Inflammatory Diseases
    Autoimmune and inflammatory conditions
  • torudokimabZB-880 / IL-33 / Monoclonal antibody / Autoimmune and Inflammatory Diseases
    Autoimmune and inflammatory conditions
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  • ABS-201ABS-201 / PRLR / anti-PRLR antibody / Cytokine biology
    Androgenetic alopeciaPhase 1/2aactiveEndometriosisPhase 1active
  • ABS-202ABS-202 / PRLR / anti-PRLR antibody / Immunology & inflammation
    Undisclosed I&I indicationCandidateactive
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  • ImdusiranAB-729; IDR / HBV viral antigens/HBV RNA transcripts / GalNAc-conjugated RNAi therapeutic; subcutaneous / Infectious disease; hepatitis B
    Chronic hepatitis B (cHBV); IM-PROVE I: imdusiran + peg-IFNα-2a + NAPhase 2aclinicalChronic hepatitis B (cHBV); IM-PROVE II: imdusiran + nivolumab + NA + VTP-300Phase 2aclinical
  • AB-101AB-101 / PD-L1 / Oral PD-L1 inhibitor / Infectious disease; hepatitis B
    Chronic hepatitis B (cHBV); AB-101-001 in healthy subjects and cHBV patientsPhase 1a/1bclinical; Part 3 dosing in cHBV patients
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  • Prulacabtagene leucelprula-cel; formerly ADI-001 / CD20 / Allogeneic gamma delta CAR T cell therapy / Autoimmune diseases
    Lupus nephritisPhase 1activeSystemic lupus erythematosusPhase 1activeSystemic sclerosisPhase 1active
  • ADI-212ADI-212 / PSMA / Next-generation gene-edited and armored allogeneic CAR gamma delta T cell therapy / Oncology
    Metastatic castration-resistant prostate cancerPreclinicalactive
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  • AKY-1189AKY-1189 / Nectin-4 / miniprotein radioconjugate; 225Ac targeted alpha radiopharmaceutical / Oncology
    Nectin-4 expressing solid tumorsPhase 1bongoingUSLocally advanced or metastatic urothelial cancer, breast cancer, NSCLC, colorectal cancer, cervical cancer, head and neck cancerPhase 1bongoingUS
  • AKY-2519AKY-2519 / B7-H3 (CD276) / miniprotein radioconjugate; 225Ac targeted alpha radiopharmaceutical; 64Cu imaging pair / Oncology
    Metastatic castration-resistant prostate cancerPhase 1bongoingUSB7-H3 expressing solid tumors, including lung and colorectal cancerPhase 1bplannedUS
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  • AKTX-101AKTX-101 / TROP2 / Antibody-drug conjugate with PH1 RNA splicing modulator payload / Oncology
    TROP2-expressing solid tumorsPreclinical / IND-enablingPhase 1 planned late 2026 or early 2027
  • AKTX-102AKTX-102 / CEACAM5 / Antibody-drug conjugate with PH1 RNA splicing modulator payload / Oncology
    CEACAM5-expressing solid tumorsDiscoveryAdvancing
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  • evorpaceptALX148 / CD47 / CD47-targeted investigational therapy / Oncology
    ENHERTU-experienced HER2-positive breast cancer; with trastuzumab + chemotherapy (ASPEN-Breast)Phase 2FPI mid-year 2025Relapsed or refractory multiple myeloma; with SARCLISA + dexamethasonePhase 1FPI Q3 2024, currently enrolling2L/3L advanced HER2-overexpressing gastric/GEJ cancer; with trastuzumab, CYRAMZA + paclitaxel (ASPEN-06)Phase 2Completed, established POC, seeking partner ex-US
  • ALX2004ALX2004 / EGFR / ADC / Oncology
    EGFR-expressing solid tumorsPhase 1FPI mid-year 2025
2assets
  • AL001AL001 / LISPRO / ionic cocrystal of lithium, proline and salicylate / Neurology/Psychiatry
    Alzheimer's diseasePhase 2clinical developmentUSBipolar disorderPhase 2clinical developmentUSMajor depressive disorderPhase 2anticipated initiation in 2026US
  • ALZN002ALZN002 / E22W / beta-amyloid / autologous mutant-peptide sensitized dendritic-cell therapeutic vaccine / Neurology
    Mild to moderate dementia of the Alzheimer's typePhase 1/2apaused in 2024; expected to resume by mid-2026US
2assets
  • dostarlimabJemperli / PD-1 / antibody / Oncology
    Endometrial cancerApprovedapproveddMMR pan tumorsApprovedapprovedRectal cancerbeing studied
  • imsidolimabIL-36R / antibody / Dermatology/Inflammation
    Generalized pustular psoriasisBLA submitted/acceptedFDA target action date December 12, 2026US
2assets
  • EpetraboroleLeucyl-tRNA synthetase / oral boron-based small molecule antibiotic / Hematology; Infectious disease
    Phlebotomy-dependent polycythemia veraPhase 2proof-of-concept studyMycobacterium abscessus complex lung diseasePhase 2IND cleared; investigator-initiated studyU.S.MelioidosisPhase 2 planneddevelopment
  • AN2-502998AN2-502998 / CPSF3 / oral boron-based benzoxaborole small molecule / Infectious disease
    Chagas diseasePhase 1first-in-human trial underway
2assets
  • tenapanorIBSRELA / small molecule / Gastrointestinal
    Chronic Idiopathic Constipation (CIC)Phase III
  • RDX10531RDX10531 / NHE3 / small molecule / Multiple therapeutic areas
    UndisclosedPreclinical
2assets
  • bemnifosbuvir/ruzasvir fixed-dose combinationBEM/RZR / HCV NS5B polymerase; HCV NS5A / oral small-molecule antiviral regimen / viral hepatitis
    hepatitis C virus (HCV) infectionPhase 3activeglobal
  • AT-587AT-587 / hepatitis E virus (HEV) / oral nucleotide analog / viral hepatitis
    chronic hepatitis E virus (HEV) infection in immunocompromised patientspreclinicalPhase 1 anticipated mid-2026
2assets
  • LarsucosterolDUR-928 / Epigenetic modulator / Hepatology
    Alcohol-associated hepatitisPhase 3Registrational Phase 3 initiated in early 2026US
  • AmiselimodMT-1303 / S1P receptor / Oral S1P modulator / Gastroenterology
    Mild-to-moderate ulcerative colitisPhase 2Internal review of opportunity ongoing
2assets
  • BezisterimNE3107 / ERK; NF-κB; TNF-α / oral small molecule anti-inflammatory insulin sensitizer / neurology; neurodegenerative disease
    Alzheimer’s diseasePhase 2/3 conductedclinicalParkinson’s diseasePhase 2SUNRISE-PD last patient evaluation completed; topline targeted Q3 2026USLong COVID neurological symptomsPhase 2ADDRESS-LC enrollingUS
  • BIV201BIV201 / continuous infusion formulation of terlipressin / liver disease
    ascites due to liver cirrhosisPhase 3 planningclinical developmentUSrefractory ascitesPhase 2b completedclinical developmentUS
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  • GLIX1GLIX1 / DNA damage response; restores TET2 activity / oral small molecule / Oncology
    GlioblastomaPhase 1Other cancersPreclinical
  • MotixafortideBL-8040 / APHEXDA / CXCR4 / peptide CXCR4 inhibitor / Oncology; rare diseases; stem cell mobilization
    Pancreatic cancerPhase 2Stem cell mobilization in multiple myelomaApprovedApproved in US; bridging study in AsiaUSAsiaSickle cell disease stem cell mobilizationPhase 1/2
2assets
  • TinlarebantLBS-008 / RBP4 / Oral small-molecule RBP4 antagonist / Ophthalmology / retinal degenerative diseases
    Stargardt disease type 1 (STGD1)Phase 3; Phase 2/3Ongoing clinical evaluation; rolling NDA submission initiated in U.S.U.S.EuropeJapanGeographic atrophy (GA) in advanced dry AMDPhase 3Ongoing clinical evaluation
  • LBS-009LBS-009 / RBP4 / Oral small-molecule RBP4 antagonist / Metabolic disease / liver disease
    Non-alcoholic fatty liver disease (NAFLD)PreclinicalPreclinical developmentNon-alcoholic steatohepatitis (NASH)PreclinicalPreclinical developmentType 2 diabetes (T2D)PreclinicalPreclinical development
2assets
  • IcovamenibMenin / Oral small molecule menin inhibitor / Diabetes; metabolic disease
    Type 1 diabetes mellitusPhase IIOngoingInsulin-deficient type 2 diabetesPhase IIEnrollingType 2 diabetes uncontrolled with a GLP-1 RA-based therapyPhase IIEnrolling
  • BMF-650BMF-650 / GLP-1 receptor / Oral small molecule GLP-1 receptor agonist / Obesity; metabolic disease
    Healthy participants / obesity and metabolic disordersPhase IOngoing
2assets
  • CAN-2409CAN-2409 / Adenovirus platform viral immunotherapy / Oncology
    Borderline resectable pancreatic adenocarcinomaPhase IIFast Track Designation (FDA); enrollment paused subject to additional fundingUSNSCLC + PD-1/PD-(L)1Phase IIFast Track Designation (FDA)USLocalized intermediate/high-risk prostate cancerBLA ReadinessFast Track Designation (FDA); Special Protocol Assessment (FDA)US
  • CAN-3110CAN-3110 / HSV platform viral immunotherapy / Oncology
    Recurrent high-grade glioma / recurrent GBMPhase IFast Track Designation (FDA)US
2assets
  • BarzolvolimabCDX-0159 / KIT / KIT antagonist mAb / Immunology/inflammation
    Chronic spontaneous urticariaPhase 3ongoingCold urticariaPhase 3ongoingSymptomatic dermographismPhase 3ongoing
  • CDX-622CDX-622 / TSLP; SCF / Bispecific antibody / Immunology/inflammation
    Healthy volunteersPhase 1ongoingAsthma proof of mechanismPhase 1ongoing
2assets
  • COMP360COMP360 / synthetic psilocybin treatment / Mental health
    Treatment-resistant depressionPhase 3NDA rolling submission underwayUSUKPost-traumatic stress disorderPhase 2b/3IND accepted; trial initiation enabledUSAnorexia nervosaPhase 2
  • Prodrug programmeprodrug programme
2assets
  • CRB-701CRB-701 / Nectin-4 / antibody-drug conjugate / Oncology
    Nectin-4 expressing solid tumorsPhase 3 pendingCSPC China: Phase 3 in cervical cancer milestoneChinaHNSCCPhase 1 in progressDose optimization; FDA Fast Track DesignationUSEuropeCervical cancerPhase 1 in progressDose optimization; FDA Fast Track DesignationUSEurope
  • CRB-913CRB-913 / CB1R / peripherally restricted oral CB1 inverse agonist / Obesity/metabolic disease
    Obesity and related conditionsPhase 1 in progress12-week dose-range study in obesity planned mid-2026
2assets
  • Vispacabtagene regedleucelCB-010 / vispa-cel / CD19 / Allogeneic anti-CD19 CAR-T cell therapy; PD-1 knockout / Hematologic malignancies
    Relapsed or refractory B cell non-Hodgkin lymphomaPhase 1Ongoing
  • CB-011CB-011 / BCMA / Allogeneic anti-BCMA CAR-T cell therapy; B2M knockout and B2M–HLA-E insertion / Hematologic malignancies
    Relapsed or refractory multiple myelomaPhase 1Ongoing
2assets
  • VIASKIN® Peanut PatchDBV712 / Peanut allergen extract / Epicutaneous immunotherapy patch; drug-device combination / Food allergy
    Peanut allergy; children 4–7 years oldPhase 3 completed; BLA submission anticipated 1H 2026Primary endpoint met in VITESSE Phase 3USPeanut allergy; toddlers 1–3 years oldPhase 3 / BLA submission anticipated 2H 2026Accelerated Approval pathway; COMFORT Toddlers safety trial ongoingUSPeanut allergy; children 1–7 years oldMAA planningEMA guidance received for circular patchEU
  • VIASKIN® Milk PatchDBV135 / Milk allergen / Epicutaneous immunotherapy patch; drug-device combination / Food allergy / immunology
    Cow’s milk allergy; ages 2–17 yearsPhase 2MILES study referencedMilk-induced eosinophilic esophagitis; ages 4–17 yearsPhase 2SMILEE study referenced
2assets
  • DT120 ODTDT120 / serotonin receptors / Orally disintegrating tablet formulation of lysergide (LSD) D-tartrate / Psychiatry / brain health
    Generalized anxiety disorderPhase 3Two Phase 3 registrational trials ongoing/evaluating efficacy and safetyU.S.EuropeMajor depressive disorderPhase 3Emerge Phase 3 ongoing; Ascend Phase 3 expected to initiate mid-2026U.S.
  • DT402DT402 / serotonergic system / R-enantiomer of MDMA (R(-)-MDMA) / Neurodevelopmental disorders
    Autism spectrum disorderPhase 2aPhase 2a study initiated after completion of Phase 1 single-ascending dose study
2assets
  • DM199 (rinvecalinase alfa)DM199 / KLK1 / Recombinant human tissue kallikrein-1 protein (rhKLK1) / Neurological
    Acute Ischemic StrokePhase 2/3ReMEDy2 Study; in progress
  • DM199 (rinvecalinase alfa)DM199 / KLK1 / Recombinant human tissue kallikrein-1 protein (rhKLK1) / Pregnancy Complications
    PreeclampsiaPhase 2Part 1A Dose-Selection / Part 1B Expansion Cohort; in progressPreeclampsiaPhase 2Parts 2 & 3 Expected Management Study; in progressFetal Growth RestrictionPhase 2Part 3 Fetal Growth Restriction; in progress
2assets
  • ClaseprubartDNTH103 / active C1s / long-acting monoclonal antibody / autoimmune / neuromuscular disorders
    Generalized Myasthenia Gravis (gMG)Phase IIPhase 2 data reported; Phase 3 trial planned mid-2026Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Phase IIICAPTIVATE; interim responder analysis completed and GO decision announced Q1 2026Multifocal Motor Neuropathy (MMN)Phase IITop-line Phase 2 data expected 2H 2026
  • DNTH212DNTH212 / LBL-047 / BDCA2 and BAFF/APRIL / extended half-life bifunctional fusion protein / autoimmune disorders
    Systemic lupus erythematosus (SLE)Phase ITwo-part Phase 1 study in China ongoing; healthy-volunteer results expected 2H 2026China
2assets
  • PBGENE-HBVPBGENE-HBV / HBV cccDNA and integrated HBV DNA / in vivo ARCUS gene editing therapy / Infectious disease
    Chronic hepatitis BPhase 1/2aELIMINATE-B trial ongoingGlobalUnited States
  • PBGENE-DMDPBGENE-DMD / Dystrophin gene exons 45-55 region / in vivo ARCUS gene editing therapy / gene excision / Neuromuscular disease
    Duchenne muscular dystrophyPhase 1/2FUNCTION-DMD study; first clinical trial site activated and enrollment begun per company IR overviewUnited States
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  • EP-104GIEP-104GI / long-acting controlled-release fluticasone propionate formulation; intra-esophageal injection / Gastrointestinal / inflammatory disease
    Eosinophilic esophagitis (EoE)Phase 1b/2Ongoing
  • EP-104IAREP-104IAR / long-acting controlled-release fluticasone propionate formulation; intra-articular injection / Pain / osteoarthritis
    Pain due to knee osteoarthritisPhase 2Completed
2assets
  • EVO756EVO756 / MRGPRX2 / Oral small-molecule antagonist / Chronic inflammatory diseases
    Chronic Spontaneous UrticariaPhase 2Atopic DermatitisPhase 2MigrainePhase 1
  • EVO301EVO301 / IL-18 / Long-acting injectable SAFA-IL-18BP fusion protein / Chronic inflammatory diseases
    Atopic DermatitisPhase 2Ulcerative ColitisPhase 1
2assets
  • DURAVYUEYP-1901 / DURAVYU / VEGF receptors; JAK1; PDGFR / vorolanib intravitreal insert; tyrosine kinase inhibitor in Durasert E bioerodible sustained-release insert / Retinal diseases
    Wet age-related macular degenerationPhase 3Trial underway; LUGANO and LUCIA fully enrolled; LUGANO topline data expected mid-2026, LUCIA to closely followGlobalDiabetic macular edemaPhase 3COMO and CAPRI pivotal trials underway; enrollment completion expected 3Q 2026Global
  • Undisclosed candidatesDurasert E programs / Retinal diseases
    Retinal diseasesPre-clinicalIn progress
2assets
  • FID-007FID-007 / Cytotoxic / Nanoencapsulated paclitaxel / Oncology
    Head and Neck (H&N) cancer, 2nd linePhase 2Ongoing; interim findings expected June 2026Ampullary or ICI resistant cancerGo/no-go based on H&N study
  • FID-022FID-022 / Cytotoxic / Oncology
    Colon cancerPhase 1Ongoing; dosing for 3rd dose level ongoingPancreatic cancerPhase 1Ongoing; dosing for 3rd dose level ongoingOvarian cancerPhase 1Ongoing; dosing for 3rd dose level ongoing
2assets
  • RevitaDuodenal mucosa / Endoscopic hydrothermal ablation / Obesity
    Weight maintenance post-GLP-1 in obesityPivotalREMAIN-1 randomization completed; 6-mo topline expected early Q4 2026US
  • RejuvaRJVA-001 / Pancreatic beta cells / AAV9 gene therapy / Type 2 Diabetes
    Type 2 diabetesPhase 1/2CTA submitted; FIH dosing & preliminary data expected H2 2026 pending approvalEUAustralia
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  • FG-3246FOR46 / CD46 / Antibody-Drug Conjugate (ADC) / Oncology
    Metastatic Castration-Resistant Prostate Cancer (mCRPC)Phase 2OngoingUnited States
  • roxadustatFG-4592 / HIF-PH / Small Molecule / Rare Disease
    Anemia associated with lower-risk myelodysplastic syndromes (LR-MDS)Phase 3PlannedUnited States
2assets
  • YUTREPIAtreprostinil inhalation powder
  • L606liposomal treprostinil inhalation suspension
2assets
  • rondecabtagene autoleucelLYL314 / Ronde-cel / CD19 / CD20 / CAR T-cell therapy / Oncology
    Relapsed/Refractory Large B-cell Lymphoma (LBCL) - 2LPhase 3ActiveUSRelapsed/Refractory Large B-cell Lymphoma (LBCL) - 3L+PivotalActiveUS
  • LYL273LYL273 / GCC (Guanylyl Cyclase-C) / CD19 / CAR T-cell therapy / Oncology
    Refractory Metastatic Colorectal Cancer (mCRC)Phase 1ActiveUS
2assets
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  • MT-601neldaleucel / Survivin, PRAME, NY-ESO-1, MAGE-A4, SSX2, WT-1 / T Cell Therapy (MAR-T cell) / Oncology
    LymphomaPancreatic Cancer
  • MT-401-OTSzelenoleucel / Multi-Tumor Associated Antigens / Allogeneic T Cell Therapy / Oncology
    Acute Myeloid Leukemia (AML)Myelodysplastic Syndrome (MDS)
2assets
  • ObicetrapibAMG-899 / CETP / Small Molecule / Cardiometabolic
    Primary hypercholesterolemia (HeFH and non-familial or mixed dyslipidemia)RegistrationOngoingGlobalEuropeAtherosclerotic Cardiovascular Disease (ASCVD)Phase 3OngoingGlobal
  • Obicetrapib / Ezetimibe FDCCETP / NPC1L1 / Small Molecule / Cardiometabolic
    Primary hypercholesterolemia (HeFH and non-familial or mixed dyslipidemia)RegistrationOngoingGlobalEurope
2assets
  • NUC-3373NUC-3373 / Thymidylate Synthase (TS) / Nucleoside analog (ProTide) / Oncology
    Advanced solid tumorsColorectal cancerLung cancer
  • NUC-7738NUC-7738 / RNA polyadenylation / Nucleoside analog (ProTide) / Oncology
    Advanced solid tumorsMelanoma
2assets
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  • NRX-101NMDA and 5-HT2A receptors / Small molecule / Central Nervous System
    Bipolar Depression with Acute Suicidal Ideation and Behavior (ASIB)Phase 3ActiveUnited StatesBipolar Depression with Subacute Suicidal Ideation and Behavior (SSIB)Phase 2/Phase 3ActiveUnited StatesDepression and Suicidality (in association with Transcranial Magnetic Stimulation)Phase 3ActiveUnited States
  • NRX-100KETAFREE / NMDA receptor / Small molecule / Central Nervous System
    Acute Suicidal Depression / Suicidal Ideation in DepressionRegistrationActiveUnited States
2assets
  • SIM0505SIM0505 / CDH6 / Antibody-drug conjugate (ADC) / Oncology
    Solid tumorsOvarian cancerLung cancer
  • LNCB74LNCB74 / B7-H4 / Antibody-drug conjugate (ADC) / Oncology
    Solid tumorsBreast cancerOvarian cancer
2assets
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  • RinzimetostatORIC-944 / PRC2 (EED subunit) / Small molecule / Oncology
    Metastatic Castration-Resistant Prostate Cancer (mCRPC)Phase 1bActiveGlobal
  • EnozertinibORIC-114 / EGFR / HER2 (exon 20 and PACC/atypical mutations) / Small molecule / Oncology
    Non-Small Cell Lung Cancer (NSCLC)Phase 1bActiveUnited StatesAustraliaCanadaHong KongMalaysiaPolandSouth KoreaSpainTaiwanUnited Kingdom
2assets
  • ORKA-001ORKA-001 / IL-23p19 / Monoclonal Antibody / Dermatology / Immunology
    Plaque PsoriasisPhase 2ActiveUnited States
  • ORKA-002ORKA-002 / IL-17A/F / Monoclonal Antibody / Dermatology / Immunology
    Plaque PsoriasisPhase 1ActiveUnited StatesHidradenitis SuppurativaPhase 1ActiveUnited States
2assets
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  • Micvotabart pelidotinMICVO, PYX-201 / EDB+FN / Antibody-Drug Conjugate (ADC) / Oncology
    Solid TumorsRecurrent and Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)
  • PYX-106PYX-106 / Siglec-15 / Monoclonal Antibody (mAb) / Oncology
    Solid Tumors
2assets
  • Lucid-MSLucid-21-302 / Demyelination / Small Molecule (NCE) / Neurology
    Multiple Sclerosis
  • FSD202FSD-202 / Mast cell activation / Small Molecule / Immunology / Pain
    Idiopathic Mast Cell Activation Syndrome (MCAS)
2assets
2assets
  • vusolimogene oderparepvecRP1 / HSV-1 oncolytic virus encoding GALV-GP R-protein and GM-CSF / Oncolytic Virus / Oncology
    Advanced MelanomaPhase 3Complete Response LetterUSNon-melanoma Skin Cancers (CSCC, MCC, BCC, Angiosarcoma)Phase 2ActiveUS
  • RP2RP2 / Oncolytic virus / Oncolytic Virus / Oncology
    Metastatic Uveal MelanomaPhase 2ActiveUSHepatocellular CarcinomaPhase 2ActiveUS
2assets
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  • ErsodetugRZ358 / Insulin receptor / Monoclonal antibody / Endocrinology/Rare Diseases
    Congenital hyperinsulinismTumor hyperinsulinism
  • RZ402RZ402 / Plasma kallikrein / Small molecule / Ophthalmology
    Diabetic macular edema (DME)Diabetic retinopathy (DR)
2assets
  • SC451SC451 / Pancreatic islet cell replacement / HIP-modified stem-cell-derived pancreatic islet cell therapy / Endocrinology / Autoimmune
    Type 1 diabetes
  • SG293SG293 / B-cells / In vivo CAR-T therapy / Oncology / Autoimmune
    B-cell cancersB-cell mediated autoimmune diseases
2assets
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2assets
  • RevumenibSNDX-5613 / Revuforj / Menin / Small Molecule / Oncology
    R/R acute leukemia with KMT2A translocation (AUGMENT-101-2A)ApprovedApprovedUSR/R NPM1m AML (AUGMENT-101-2C)ApprovedApprovedUSFrontline unfit NPM1m and KMT2Ar acute leukemias (EVOLVE-2)Phase 3ActiveUS
  • AxatilimabSNDX-6352 / Niktimvo / CSF-1R / Monoclonal Antibody / Oncology / Immunology
    Refractory Chronic GVHD (AGAVE-201)ApprovedApprovedUSFrontline Chronic GVHD (corticosteroids combination)Phase 3ActiveUSFrontline Chronic GVHD (ruxolitinib combination)Phase 2ActiveUS
2assets
  • Tralesinidase AlfaTA-ERT / Alpha-N-acetylglucosaminidase (NAGLU) / Enzyme Replacement Therapy / Neurology / Rare Disease
    Mucopolysaccharidosis Type IIIB (MPS IIIB) / Sanfilippo Syndrome Type BPhase 3ActiveGlobal
  • TildacerfontCorticotropin-releasing factor type-1 (CRF1) receptor / Small Molecule / Neurology / Psychiatry
    Major Depressive Disorder (MDD)Phase 2ActiveEurope
2assets
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  • SL-325SL-325 / DR3 (Death Receptor 3) / Monoclonal Antibody / Inflammatory and Immune-Mediated Diseases
    Inflammatory Bowel Disease (IBD)Crohn's DiseaseUlcerative Colitis
  • SL-425SL-425 / DR3 (Death Receptor 3) / Monoclonal Antibody / Inflammatory and Immune-Mediated Diseases
    Inflammatory Bowel Disease (IBD)
2assets
  • TARA-002TARA-002 / Broad immunopotentiator / Cell therapy / Oncology / Rare Diseases
    Non-Muscle Invasive Bladder Cancer (NMIBC)Phase 2ActiveUnited StatesLymphatic Malformations (LMs)Phase 2ActiveUnited States
  • IV Choline ChloridePhospholipid substrate replacement / Small molecule / Intravenous replacement therapy / Rare Diseases
    Patients dependent on long-term parenteral support (PS) / Choline deficiencyPhase 3ActiveUnited States
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  • Oxylanthanum CarbonateOLC / Renazorb / Phosphate / Small molecule / Nephrology
    Hyperphosphatemia in chronic kidney disease (CKD) on dialysisNDAResubmittedUnited States
  • UNI-494UNI-494 / KATP channel / Nitric oxide (nicorandil prodrug) / Small molecule / Nephrology
    Acute Kidney Injury (AKI)Phase 1CompletedUnited KingdomUnited StatesPrevention of Delayed Graft Function (DGF) in kidney transplant patientsPhase 1ActiveUnited States
2assets
2assets
  • cantharidinVP-102 / YCANTH / Unknown / Small Molecule / Dermatology
    Molluscum ContagiosumApprovedActiveCommon WartsPhase 3ActiveExternal Genital WartsPhase 2Active
  • VP-315VP-315 / Oncolytic peptide / Peptide / Dermatologic Oncology
    Basal Cell CarcinomaPhase 2ActiveSquamous Cell CarcinomaPhase 1/2Active
2assets
  • VAR 200Cholesterol Efflux Pathway (ABCA1 and ABCG1) / Cholesterol Efflux Mediator / Renal Diseases
    Focal Segmental Glomerulosclerosis (FSGS)Phase 2activeUnited StatesDiabetic Kidney Disease (DKD)Phase 2activeUnited StatesAlport SyndromePreclinicalactiveUnited States
  • IC 100Inflammasome ASC / Monoclonal Antibody / Inflammatory Diseases
    Cardiometabolic Conditions (Obesity-associated)PreclinicalactiveUnited StatesParkinson's DiseasePreclinicalactiveUnited StatesOrphan Renal DiseasesPreclinicalactiveUnited States
1assets
  • ObefazimodABX464 / microRNA-124 (miR-124) enhancer / Oral small molecule / Inflammatory diseases
    Moderately to severely active ulcerative colitisPhase 3ABTECT induction data readout completed July 2025; maintenance data expected Q2 2026Crohn's diseasePhase 2bENHANCE-CD initiated; 12-week induction data expected Q4 2026Moderately to severely active ulcerative colitis, combination therapyPreclinicalEvaluation of obefazimod combined with other agents; decision on combination agent expected in 2026
1assets
  • cytisiniclinenicotinic acetylcholine receptor / plant-derived alkaloid; partial agonist / nicotine dependence
    smoking cessation in adultsNDA accepted; PDUFA June 20, 2026under FDA reviewUSe-cigarette or vaping cessationPhase 2 completed; Phase 3 design agreedBreakthrough Therapy designation; FDA CNPVUS
1assets
  • elraglusib9-ING-41 / GSK-3β / small molecule inhibitor / Oncology
    1L metastatic pancreatic cancer with gemcitabine/nab-paclitaxelPhase 2ongoing; Actuate-1801 Part 3BUSEUCanada1L metastatic pancreatic cancer with gemcitabine/nab-paclitaxelPhase 3in planning; registration trial contingent on funding1L metastatic pancreatic cancer with FOLFIRINOXPhase 2investigator-initiated; closed to enrollment
1assets
  • loncastuximab tesirine-lpylZYNLONTA / CD19 / antibody-drug conjugate / Oncology
    LOTIS-2 in 3L+ r/r DLBCLPhase 3/ConfirmatoryLOTIS-5 in r/r DLBCLPhase 3/ConfirmatoryLOTIS-7 in r/r B-NHLPhase 1b
1assets
  • ABP-450ABP-450 / Acetylcholine release / 900 kDa botulinum toxin type A complex; biosimilar biologic / Neurology; neuromuscular disorders
    Episodic migrainePhase 2United StatesChronic migrainePhase 2United StatesCervical dystoniaPhase 2United States
1assets
  • Ampligenrintatolimod / Ampligen / TLR3 / dsRNA immuno-modulator / Oncology; immune disorders; viral diseases
    Metastatic pancreatic ductal adenocarcinomaPhase 1b/2DURIPANCAdvanced, recurrent ovarian cancerPhase 2Refractory melanomaPhase 2
1assets
  • pemvidutideALT-801 / glucagon and GLP-1 receptors / balanced 1:1 glucagon/GLP-1 dual receptor agonist peptide / serious liver diseases
    Metabolic dysfunction-associated steatohepatitis (MASH)Phase 3 plannedPERFORMA Phase 3 initiation planned 2H 2026; FDA Fast Track and Breakthrough Therapy DesignationsU.S.Alcohol use disorder (AUD)Phase 2RECLAIM Phase 2 enrollment complete; topline data expected 3Q 2026; FDA Fast Track DesignationU.S.Alcohol-associated liver disease (ALD)Phase 2RESTORE Phase 2 enrolling; enrollment completion expected 3Q 2026U.S.
1assets
  • (Z)-endoxifenestrogen receptor / oral small molecule SERM/D / Oncology; rare diseases
    Metastatic breast cancerPhase 1/2FDA pathway discussions; IND study may proceed reportedUSAdjuvant breast cancerFDA pathway discussionsUSNeoadjuvant breast cancerongoing clinical studies: EVANGELINE and I-SPY2
1assets
  • belzupacap sarotalocanbel-sar; AU-011 / tumor-associated GAGs/HSPGs / virus-like drug conjugate (VDC) / oncology
    early-stage choroidal melanomaPhase 3ongoing CoMpass trialUSEuropemetastases to the choroidPhase 2ongoingUSEuropecancers of the ocular surfacePhase 1initiating proof-of-concept trialUSEurope
1assets
  • AbdakibartAVTX-009 / IL-1β / Humanized IgG4 monoclonal antibody / Immune-mediated inflammatory diseases
    Hidradenitis suppurativaPhase 2 completed; registrational Phase 3 plannedPositive topline Phase 2 LOTUS results reported May 5, 2026
1assets
  • ficerafusp alfaBCA101 / EGFR/TGF-β / bifunctional EGFR-directed monoclonal antibody bound to a human TGF-β ligand trap / Oncology
    1L recurrent/metastatic HPV-negative head and neck squamous cell carcinomaPhase 2/3ongoinggloballate-line MSS RAS wild-type metastatic colorectal cancerPhase 1/1benrolling expansion cohort
1assets
  • AMELUZ®AMELUZ® / aminolevulinic acid HCl topical gel; photodynamic therapy / Dermatology
    New formulationMild to moderate actinic keratosis: face & scalp larger treatment area (3 tubes)Superficial basal cell carcinomaPhase 3database lockedUS
1assets
  • BB-301BB-301 / PABPN1 / AAV ddRNAi silence-and-replace genetic medicine / Rare disease; neuromuscular
    Oculopharyngeal muscular dystrophy (OPMD)Phase 1b/2aClinical-stage
1assets
  • rese-celCABA-201 / CD19 / Autologous fully human 4-1BB CD19-CAR T cell therapy / Autoimmune disease
    DermatomyositisRegistrationalrecruitingUSAntisynthetase syndromeRegistrationalrecruitingUSImmune-mediated necrotizing myopathyPhase 1/2recruitingUS
1assets
  • gedatolisibPI3K/AKT/mTOR pathway; class I PI3K isoforms; mTORC1/2 / small molecule kinase inhibitor / Oncology
    HR+/HER2- advanced breast cancer, 2nd linePhase 3; NDA accepted/Priority Review for PIK3CA WTVIKTORIA-1 reported results; PDUFA July 17, 2026GlobalHR+/HER2- locally advanced or metastatic breast cancer, 1st linePhase 3VIKTORIA-2 ongoingNorth AmericaEuropeLatin AmericaAsiaMetastatic castration-resistant prostate cancerPhase 1/2CELC-G-201 ongoingUnited StatesEurope
1assets
  • Cretostimogene grenadenorepvecCG0070 / Intravesical oncolytic immunotherapy / Bladder cancer
    High-risk BCG-unresponsive NMIBC with CIS with or without high-grade Ta/T1 disease; monotherapy; BOND-003 Cohort CPhase 3Enrollment complete; data presented at AUA 2025High-risk BCG-unresponsive NMIBC with high-grade Ta/T1; monotherapy; BOND-003 Cohort PPhase 2Enrollment complete; topline results at SUO 2025Intermediate-risk NMIBC; monotherapy; PIVOT-006Phase 3Enrollment complete; topline data expected 1H 2026
1assets
  • RademikibartCBP-201 / IL-4Rα / human monoclonal antibody / Inflammatory diseases
    Acute exacerbations of COPDPhase 2ongoingGlobalAcute exacerbations of asthmaPhase 2ongoingGlobalChronic asthmaongoing studies by SimcereChina
1assets
  • onvansertibPLK1 / oral small-molecule PLK1 inhibitor / Oncology
    1L RAS-mutated metastatic colorectal cancerPhase 2randomized CRDF-0042L KRAS-mutated metastatic colorectal cancerPhase 1b/2completed2L metastatic colorectal cancerPhase 3ONSEMBLE/CRDF-003 randomized
1assets
  • EDIT-401EDIT-401 / LDLR / in vivo CRISPR gene editing medicine / Cardiovascular diseases
    Hyperlipidemia / LDL-C reductionPreclinicalIND/CTA submission planned by mid-2026; human proof-of-concept targeted by end of 2026Atherosclerotic cardiovascular disease (ASCVD)PreclinicalIn developmentHeterozygous familial hypercholesterolemia (HeFH)PreclinicalFirst-in-human trial planned in 2026
1assets
  • FB102FB102 / CD122 / monoclonal antibody / Autoimmune and autoimmune-related diseases
    Celiac diseasePhase 2Topline results expected in 2026; U.S. arm included after FDA IND approvalUnited StatesVitiligoPhase 1bTopline results expected in 1H 2026Alopecia areataPhase 1bData readout expected in 2026
1assets
  • PEDMARKPEDMARK / PEDMARQSI / cisplatin-induced ototoxicity / small molecule; sodium thiosulfate injection / oncology supportive care
    Reduce risk of ototoxicity associated with cisplatin in pediatric patients 1 month and older with localized, non-metastatic solid tumorsApprovedMarketed; ongoing evidence generationUSEUUKCisplatin-induced ototoxicity in pediatric and AYA patients with non-metastatic solid tumorsPhase 2/3Positive topline results; pursuing registrationJapanOtoprotection in adults receiving cisplatin chemotherapy, including head and neck and testicular cancersPhase I/IIInvestigator-sponsored study initiatedUS
1assets
  • pociredirFTX-6058 / EED / PRC2; HbF induction / oral small molecule / Hematology
    Sickle cell diseasePhase 1bactive; potential registration-enabling trial planned for 2H 2026United States
1assets
  • imetelstatRYTELO / telomerase / telomerase inhibitor / Hematologic malignancies
    JAK inhibitor relapsed/refractory myelofibrosisPhase 3open for screening and enrollmentfrontline myelofibrosis, intermediate-1/intermediate-2/high-risk; combination with ruxolitinibPhase 1open for screening and enrollmentrelapsed/refractory/intolerant acute myeloid leukemia or high-risk MDS after hypomethylating agentsPhase 2investigator-led; open for screening and enrollment
1assets
  • GLSI-100GP2 + GM-CSF / HER2/neu / peptide immunotherapy vaccine / Oncology
    Prevention of recurrence in HER2/neu-positive breast cancer after surgery and trastuzumab-based therapyPhase 3Ongoing; Fast Track designated; FLAMINGO-01United StatesEurope
1assets
  • Olvi-Vecolvimulogene nanivacirepvec / Oncolytic vaccinia virus immunotherapy / Oncology
    Platinum-resistant/refractory ovarian cancer (PRROC)Phase 3Enrolling; topline data expected 2H 2026 (OnPrime/GOG-3076; NCT05281471)USNon-small cell lung cancer (NSCLC), advanced/metastatic recurrentPhase 2Dose-escalation ongoing (VIRO-25; NCT06463665); systemic IV Olvi-Vec + platinum-doublet + ICI vs docetaxelUSSmall cell lung cancer (SCLC), platinum-relapsed/refractoryPhase 1b/2Dose selection ongoing (OLVI-VEC-SCLC-202; NCT07136285); Olvi-Vec + platinum + etoposideChinaUS
1assets
  • NXC-201NXC-201 / BCMA / sterically-optimized autologous CAR-T / Hematology
    Relapsed/refractory AL AmyloidosisPhase 2BLA-enabling; enrollment completedUSIsraelSelect immune-mediated diseasesExpanding
1assets
  • TH103TH103 / VEGF / HSPG / Recombinant fusion protein / Retinal Diseases
    Neovascular age-related macular degeneration (nAMD)Phase 1b/2OngoingDiabetic eye diseasePlannedPlannedRetinal vein occlusionPlannedPlanned
1assets
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  • LB-100LB-100 / Protein Phosphatase 2A (PP2A) / Small Molecule / Oncology
    Ovarian Clear Cell CarcinomaPhase 2ActiveUnited StatesMetastatic Colon CancerPhase 1bActiveGlobalAdvanced Soft Tissue SarcomaPhase 1/2PausedEurope
1assets
1assets
  • SonelokimabM1095 / IL-17A and IL-17F (tri-specific Nanobody) / Nanobody (VHH) / Immunology (Dermatology and Rheumatology)
    Hidradenitis Suppurativa (HS)Phase 3OngoingGlobalAdolescent Hidradenitis Suppurativa (Teen HS)Phase 3OngoingGlobalPsoriatic Arthritis (PsA)Phase 3OngoingGlobal
1assets
  • lorundrostatMLS-101 / Aldosterone synthase (CYP11B2) / Small molecule / Cardio-Renal-Metabolic Syndrome
    Uncontrolled and treatment-resistant hypertensionPhase 3CompletedGlobalObstructive Sleep Apnea (OSA) and HypertensionPhase 2OngoingGlobalChronic Kidney Disease (CKD)Phase 2OngoingGlobal
1assets
  • NBTXR3JNJ-1900 / Hensify / Tumor cell (Radioenhancer) / Functionalized hafnium oxide (HfO2) nanoparticles / Oncology
    Locally Advanced Head and Neck Squamous Cell Carcinoma (LA-HNSCC) - Platinum-ineligiblePhase 3OngoingGlobalRecurrent/Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)Phase 1/2OngoingGlobalSoft Tissue SarcomaApprovedMarketedEurope
1assets
  • NKX019NKX019 / CD19 / Allogeneic CAR-NK Cell Therapy / Autoimmune Diseases
    Lupus NephritisSystemic SclerosisInflammatory Myopathy
1assets
  • Tumor Treating FieldsTTFields / Optune Gio, Optune Lua, Optune Pax, Optune Mya / Mitotic spindle / Electric Field Therapy / Oncology
    Glioblastoma (GBM)ApprovedMarketedUnited StatesEuropeJapanGlioblastoma (GBM) - Earlier use (TRIDENT)Phase 3ActiveGlobalNon-Small Cell Lung Cancer (NSCLC)ApprovedMarketedUnited StatesJapanEurope
1assets
  • PALI-2108PALI-2108 / PDE4 / Small Molecule Prodrug / Gastroenterology
    Ulcerative Colitis (UC)Phase 2ActiveGlobalCrohn's Disease (CD)Phase 2ActiveGlobal
1assets
1assets
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  • NimacimabCB1 / Monoclonal antibody / Metabolic Diseases
    ObesityPhase 2activeUnited States
1assets
1assets
1assets
  • Epinephrine nasal sprayARS-1 / neffy / Alpha and beta-adrenergic receptors / Small Molecule / Immunology & Allergy
    Type I Allergic Reactions (including Anaphylaxis)ApprovedActiveUnited StatesEuropean UnionCanadaUrticaria (Acute Flares of Chronic Spontaneous Urticaria)Phase 2ActiveUnited States
1assets
  • MolgramostimMolbreevi / GM-CSF receptor / Recombinant protein (rhGM-CSF) / Respiratory / Rare Lung Diseases
    Autoimmune Pulmonary Alveolar Proteinosis (aPAP)SubmissionRegulatory SubmissionUSUKEU
1assets
  • TERN-701TERN-701 / BCR::ABL1 / Small Molecule / Oncology
    Chronic Myeloid Leukemia (CML)Phase 1/2ActiveGlobal
1assets
  • pacibekitugTOUR006 / IL-6 / Monoclonal Antibody / Cardiovascular and Immune Diseases
    Atherosclerotic Cardiovascular Disease (ASCVD)Phase 2ActiveGlobalThyroid Eye Disease (TED)Phase 2ActiveGlobalAbdominal Aortic Aneurysm (AAA)Phase 2PlannedGlobal
1assets
1assets
  • idecabtagene vicleucelAbecma / BCMA / CAR-T Cell Therapy / Oncology
    Relapsed or refractory multiple myelomaMarketedApprovedUSEU
1assets
  • verekitugUPB-101 / TSLP receptor (TSLPR) / Monoclonal antibody / Respiratory/Inflammatory Diseases
    Severe AsthmaChronic rhinosinusitis with nasal polyps (CRSwNP)Chronic obstructive pulmonary disease (COPD)
1assets
  • TelitaciceptRC18 / Taiai / BAFF/APRIL (BLyS/APRIL) / Recombinant Fusion Protein / Autoimmune Diseases
    Generalized Myasthenia Gravis (gMG)Phase 3Active / Approved in ChinaGlobalChinaSystemic Lupus Erythematosus (SLE)Phase 3Active / Approved in ChinaGlobalChinaRheumatoid Arthritis (RA)Phase 3Active / Approved in ChinaGlobalChina
1assets
  • CadisegliatinTTP399 / Liver-selective glucokinase / Small molecule / Endocrinology
    Type 1 Diabetes (adjunctive to insulin)Phase 3OngoingUnited StatesType 2 DiabetesPhase 2PlannedUnited Arab Emirates
1assets
  • AzenosertibZN-c3 / WEE1 / Small Molecule / Oncology
    Cyclin E1-positive platinum-resistant ovarian cancer (PROC)Advanced solid tumors
0assets

No disclosed assets in this row.