Pipeline Watchlist
Company Pipelines
Recent additions
14 daysCureVac N.V.
curevac.comUPEC FimH ferritin nanoparticle mRNA vaccine candidate
Infectious disease / Preclinical / urinary tract infections caused by uropathogenic E. coli
CureVac N.V.
curevac.comCOVID-19/influenza combination mRNA vaccine candidate
Infectious disease / Phase 1/2 / COVID-19 and influenza
CureVac N.V.
curevac.comavian influenza H5N1 mRNA vaccine candidate
Infectious disease / Phase 2 / avian influenza H5N1
CureVac N.V.
curevac.comseasonal influenza mRNA vaccine candidate
Infectious disease / Phase 2 / seasonal influenza
CureVac N.V.
curevac.comsqNSCLC shared-antigen cancer precision immunotherapy candidate
Oncology / IND/CTA filed / squamous non-small cell lung cancer
Crinetics Pharmaceuticals, Inc.
crinetics.comOral GIP Nonpeptide
Metabolic disease / Preclinical / Diabetes; obesity
Crinetics Pharmaceuticals, Inc.
crinetics.comOral GLP-1 Nonpeptide
Metabolic disease / Preclinical / Diabetes; obesity
Crinetics Pharmaceuticals, Inc.
crinetics.comAtumelnant
CRN04894 / Endocrinology / Phase 3 / Congenital adrenal hyperplasia (adult)
Companies
62assets
- LutatheraAAA601 / Lutathera / SSTR / Radioligand therapy / Oncology: Solid TumorsGastroenteropancreatic neuroendocrine tumorsPhase 3Supplementary; filing 2028GEPNET, pediatricsPhase 2
- 177Lu-NeoBAAA603 / GRPR / Radioligand therapy / Oncology: Solid TumorsBreast cancerPhase 1Multiple solid tumorsPhase 2Lead; filing >= 2029
- AAA614FAP / Radioligand therapy / Oncology: Solid TumorsSolid tumorsPhase 2Lead
- PluvictoAAA617 / Pluvicto / PSMA / Radioligand therapy / Oncology: Solid TumorsOligometastatic prostate cancerPhase 3Supplementary; filing >= 2029Metastatic hormone sensitive prostate cancer (mHSPC)RegistrationSupplementary
- 225Ac-PSMA-617AAA817 / PSMA / Radioligand therapy / Oncology: Solid TumorsMetastatic 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: HematologyChronic myeloid leukemia, pediatricsPhase 2Supplementary; filing 2027
- CosentyxAIN457 / Cosentyx / IL17A / Inhibitor / ImmunologyPolymyalgia rheumaticaRegistrationSupplementary; filing 2026
- AimovigAMG334 / Aimovig / CGRPR / Antagonist / In-market Brands and Global HealthMigraine, pediatricsPhase 3Supplementary; filing 2028
- AMO959DNA PK / Inhibitor / Oncology: Solid TumorsProstate cancerPhase 1Lead
- MayzentBAF312 / Mayzent / S1P1,5 receptor modulator / NeuroscienceMultiple sclerosis, pediatricsPhase 3Supplementary; filing 2027
- VijoyceBYL719 / Vijoyce / PI3K-alpha / Inhibitor / Oncology: Solid TumorsLymphatic malformationsPhase 3Supplementary; filing >= 2029
- farabursenCYX082 / MIR17 / Inhibitor / Cardiovascular, Renal and MetabolicAutosomal dominant polycystic kidney diseasePhase 1Lead
- pelabresibDAK539 / BET / Inhibitor / Oncology: HematologyMyelofibrosisPhase 3Lead; filing 2026
- DFT383CTNS gene delivery / NeuroscienceCystinosisPhase 1Lead
- DII235siRNA targeting Lp(a) MRNA / Cardiovascular, Renal and MetabolicCVRR-Lp(a)Phase 2Lead; filing >= 2029
- del-braxDWH213 / siRNA DUX4 knockdown / NeuroscienceFacioscapulohumeral muscular dystrophyPhase 3Lead; filing 2028
- tulmimetostatDZR123 / EZH1, EZH2 / Inhibitor / Oncology: Solid TumorsMetastatic prostate cancerPhase 1Solid tumors & lymphomasPhase 2Lead
- ECI830CDK2 / Inhibitor / Oncology: Solid TumorsBreast cancerPhase 1Lead
- EDI048CpPI(4)K / Inhibitor / In-market Brands and Global HealthCryptosporidiosisPhase 2Lead
- EDK060Lead Indication / NeuroscienceCharcot-Marie-Tooth diseasePhase 1
- ESP359DLL3 / Radioligand therapy / Oncology: Solid TumorsSolid tumorsPhase 1Lead
- del-desiranEWF980 / siRNA DMPK knockdown / NeuroscienceMyotonic dystrophy type 1Phase 3Lead; filing 2027
- EYU688NS4B / Inhibitor / In-market Brands and Global HealthDengue feverPhase 2Lead
- zigakibartFUB523 / Anti-APRIL / Cardiovascular, Renal and MetabolicIgA nephropathyPhase 3Lead; filing 2027
- 177Lu-NNS309FXX489 / FAP / Radioligand therapy / Oncology: Solid TumorsSolid tumorsPhase 1Lead
- GCJ904Lead Indication / Oncology: Solid TumorsSolid tumorsPhase 1
- GHZ339Lead Indication / ImmunologyAtopic dermatitisPhase 2filing >= 2029
- GIA632IL-15 mAb / ImmunologyAtopic dermatitisPhase 2Lead; filing >= 2029VitiligoPhase 2Supplementary; filing >= 2029
- GXV813M4 / Agonist / NeuroscienceSchizophreniaPhase 2Lead
- HJB647Lead Indication / Cardiovascular, Renal and MetabolicHeart failurePhase 1
- votoplamHTT227 / Huntingtin modulator / NeuroscienceHuntington’s diseasePhase 3Lead; filing >= 2029
- INE963Plasmodium falciparum / Inhibitor / In-market Brands and Global HealthMalariaPhase 2Lead
- IPX643Lead Indication / ImmunologyInflammation-driven diseasesPhase 1
- ITU512HbF inducing agent / In-market Brands and Global HealthSickle cell diseasePhase 1Lead
- luxdegalutamideJSB462 / Androgen receptor protein degrader / Oncology: Solid TumorsMetastatic hormonal sensitive prostate cancerPhase 2Metastatic castration resistant prostate cancerPhase 2Lead; filing >= 2029
- cipargaminKAE609 / PfATP4 / Inhibitor / In-market Brands and Global HealthMalaria, uncomplicatedPhase 2Supplementary; filing >= 2029Malaria severePhase 2Lead; filing >= 2029
- LeqvioKJX839 / Leqvio / siRNA (regulation of LDL-C) / Cardiovascular, Renal and MetabolicCVRR (primary prevention)Phase 3Supplementary; filing >= 2029CVRR (secondary prevention)Phase 3Supplementary; filing 2027
- Ganaplacide + lumefantrineKLU156 / Non-artemisinin plasmodium falciparum / Inhibitor / In-market Brands and Global HealthMalaria, uncomplicatedPhase 3Lead; filing 2026
- del-zotaKPE179 / PMO mediated exon 44 skipping / NeuroscienceDuchenne muscular dystrophyPhase 2Lead; filing 2026
- FabhaltaLNP023 / Fabhalta / CFB / Inhibitor / Neuroscience / Cardiovascular, Renal and Metabolic / Oncology: Hematology / OthersGeneralized myasthenia gravisPhase 3Supplementary; filing 2027C3 glomerulopathy, pediatricsPhase 3IgAN, pediatricsPhase 3
- RhapsidoLOU064 / Rhapsido / BTK / Inhibitor / ImmunologyChronic spontaneous urticaria, pediatricsPhase 3Hidradenitis suppurativaPhase 3Supplementary; filing 2027Chronic inducible urticaria (CINDU)Phase 3Supplementary; filing 2026
- remibrutinibLOU064 / BTK / Inhibitor / NeuroscienceMultiple sclerosis, secondary progressivePhase 3Supplementary; filing >= 2029Myasthenia gravisPhase 3Supplementary; filing 2028Multiple sclerosis, relapsing remittingPhase 3Supplementary; filing 2027
- LTP001SMURF1 / Inhibitor / Cardiovascular, Renal and MetabolicIdiopathic pulmonary fibrosisPhase 2Pulmonary arterial hypertensionPhase 2Lead; filing >= 2029
- LXE408Proteasome / Inhibitor / In-market Brands and Global HealthChagasPhase 2Visceral leishmaniosisPhase 2Lead; filing >= 2029
- abelacimabMAA868 / FXI / Inhibitor / Cardiovascular, Renal and MetabolicAtrial fibrillationPhase 3Lead; filing 2028
- MAS825IL18 Inhibitor, IL1B / ImmunologyStill’s diseasePhase 2Lead
- NIO752Tau antisense oligonucleotide / NeuroscienceAlzheimer’s diseasePhase 1Progressive supranuclear palsyPhase 1Lead
- OJR520Lead Indication / Cardiovascular, Renal and MetabolicCKDPhase 1
- KesimptaOMB157 / Kesimpta / CD20 / Antagonist / NeuroscienceMultiple sclerosis, new dosing regimenPhase 3Supplementary; filing 2027Multiple sclerosis, pediatricsPhase 3Supplementary; filing 2026
- PAC001Anti-IL-6 mAb / Cardiovascular, Renal and Metabolic / OthersASCVD pacibekitugPhase 2Lead; filing >= 2029Thyroid eye disease (TED) pacibekitugPhase 2
- PIT565Anti-CD19, Anti-CD2, Anti-CD3 / ImmunologySystemic lupus erythematosusPhase 1Rheumatoid arthritisPhase 1
- RydaptPKC412 / Rydapt / Multi-targeted kinase / Inhibitor / In-market Brands and Global HealthAcute myeloid leukemia, pediatricsPhase 2Supplementary; filing 2027
- PKN605HDAC6 / Inhibitor / Cardiovascular, Renal and MetabolicAtrial FibrillationPhase 2Lead
- QCZ484Lead Indication / Cardiovascular, Renal and MetabolicHypertensionPhase 2filing >= 2029
- AtecturaQMF149 / Atectura / LABA + ICS / In-market Brands and Global HealthAsthma, pediatricsPhase 3Supplementary; filing >= 2029
- AdakveoSEG101 / Adakveo / P-selectin / Inhibitor / In-market Brands and Global HealthSickle cell disease, pediatricsPhase 3Supplementary; filing >= 2029
- TIN816ATP modulator / Cardiovascular, Renal and MetabolicAcute kidney injuryPhase 2Lead
- pelacarsenTQJ230 / ASO targeting Lp(a) / Cardiovascular, Renal and MetabolicCVRR (secondary prevention) in patients with elevated Lp(a)Phase 3Lead; filing 2026
- ianalumabVAY736 / BAFF-R inhibitor, ADCC-mediated B-cell depletor / Immunology / Oncology: HematologyLupus NephritisPhase 3Supplementary; filing 2028Systemic lupus erythematosus (SLE)Phase 3Supplementary; filing 2028Systemic sclerosisPhase 2Supplementary; filing 2028
- VHB937TREM2 stabilizer and activator / NeuroscienceAmyotrophic lateral sclerosisPhase 2Lead; filing >= 2029Alzheimer's diseasePhase 2Supplementary; filing >= 2029
- YMI024Lead Indication / Cardiovascular, Renal and MetabolicCVRRPhase 1
- rapcabtagene autoleucelYTB323 / CD19 CAR-T / Immunology / Neuroscience / Oncology: HematologyRheumatoid arthritis and severe, refractory Sjögren’s diseasePhase 1Systemic sclerosisPhase 2Supplementary; filing >= 2029MyositisPhase 2Supplementary; filing >= 2029
37assets
- ABBV-8736ABBV-8736 / TREM1 / Biologic / ImmunologyCrohn's DiseasePhase 2
- TavapadonD1/D5 partial agonist / Small molecule / NeuroscienceParkinson's DiseaseSubmitted
- ABBV-525ABBV-525 / MALT1 / Small molecule / OncologyHematologic TumorsPhase 1
- Telisotuzumab vedotinABBV-399 / EMRELIS / c-Met / Antibody-drug conjugate / OncologyNon-Small Cell Lung CancerApprovedUS
- EpcoritamabGen3013 / EPKINLY/TEPKINLY / CD3, CD20 / Bispecific antibody / OncologyRelapsed/Refractory DLBCLApprovedUSEURelapsed/Refractory Follicular LymphomaApprovedRelapsed/Refractory CLLPhase 2
- ABBV-466 (Skyrizi + ABBV-382)ABBV-466 / SKYRIZI / IL-23 + anti-a4b7 mAb / Biologic combination / ImmunologyCrohn's DiseasePhase 2
- BudigalimabABBV-181 / PD1 / Biologic / OncologySolid and Hematologic TumorsPhase 2
- ABBV-701ABBV-701 / TL1A / Biologic / ImmunologyInflammatory Bowel DiseasePhase 1
- ABBV-932ABBV-932 / DRD2/DRD3 modulator / Small molecule / NeuroscienceBipolar DepressionPhase 2Generalized Anxiety DisorderPhase 2
- ABBV-9832 (Lutikizumab + Ravagalimab)ABBV-9832 / IL-1a/1b + CD40 antagonist / Biologic combination / ImmunologyRheumatoid ArthritisPhase 2
- ABBV-6628ABBV-6628 / C5 / Biologic / Eye CareGeographic AtrophyPhase 1
- ABBV-619ABBV-619 / CD19 / Biologic/CAR-T / ImmunologyAutoimmune diseasesPhase 1
- LivmoniplimabABBV-151 / GARP-TGF-beta1 / Biologic / OncologyHepatocellular CarcinomaPhase 2Solid TumorsPhase 2Non-Small Cell Lung CancerPhase 2
- Armour ThyroidArmour Thyroid / T3T4 / Biologic / Other SpecialtiesHypothyroidismPhase 3
- ABBV-319ABBV-319 / CD19 / Biologic ADC / ImmunologySjogren's diseasePhase 1Systemic Lupus ErythematosusPhase 1
- ABBV-722ABBV-722 / LPAR1 / Small molecule / ImmunologyIdiopathic Pulmonary FibrosisPhase 1Systemic SclerosisPhase 1
- ABBV-142ABBV-142 / LPAR1 / Biologic / ImmunologyIdiopathic Pulmonary FibrosisPhase 1
- ABBV-324ABBV-324 / Biologic ADC / OncologySolid TumorsPhase 1
- ABBV-295ABBV-295 / Long-Acting Amylin Analog / Large molecule / Other SpecialtiesObesityPhase 1
- SurzetoclaxABBV-453 / BCL-2 / Small molecule / OncologyRelapsed/Refractory Multiple MyelomaPhase 1
- ABBV-2002ABBV-2002 / PAR-2 / Biologic / NeuroscienceMigrainePhase 2
- IcalcaprantABBV-1354 / KOR antagonist / Small molecule / NeuroscienceBipolar DepressionPhase 2Major Depressive DisorderPhase 2
- ABBV-1042ABBV-1042 / M4 receptor / Small molecule / NeuroscienceBipolar ManiaPhase 1
- Surabgene LomparvovecABBV-RGX-314 / VEGF / Gene therapy / Eye Carewet AMD (subretinal)Phase 3wet AMD (suprachoroidal)Phase 2Diabetic Retinopathy (suprachoroidal)Phase 2
- UbrogepantUBRELVY / CGRP receptor / Small molecule / NeuroscienceMenstrual MigrainePhase 3
- ABBV-2001ABBV-2001 / BCMA, CD38, CD3 / Biologic trispecific T-cell engager / OncologyRelapsed/Refractory Multiple MyelomaPhase 1
- Pivekimab SunirinePVEK / CD123 / Biologic ADC / Oncology
- Mirvetuximab soravtansineELAHERE / FR-alpha / Biologic ADC / OncologyPlatinum-Resistant Ovarian CancerApprovedPlatinum-Sensitive Ovarian CancerPhase 3
- AtogepantQULIPTA / CGRP receptor / Small molecule / NeuroscienceAcute MigraineSubmittedOUSMenstrual MigrainePhase 3OUS
- EmraclidineM4 PAM / Small molecule / NeuroscienceSchizophreniaPhase 2PsychosisPhase 1
- UpadacitinibABT-494 / RINVOQ / JAK1 / Small molecule / ImmunologyAlopecia AreataSubmittedUSEUHidradenitis SuppurativaPhase 3Systemic Lupus ErythematosusPhase 3
- ABBV-166 (Skyrizi + Lutikizumab)ABBV-166 / SKYRIZI / IL-23 + IL-1a/1b / Biologic combination / ImmunologyPsoriatic ArthritisPhase 2
- ABBV-848ABBV-848 / IRAK4 / Small molecule / ImmunologyRheumatoid ArthritisPhase 1
- ABBV-706ABBV-706 / SEZ6 / Biologic ADC / OncologyFirst Line Small Cell Lung CancerPhase 2Relapsed/Refractory Small Cell Lung CancerPhase 3Neuroendocrine NeoplasmsPhase 1
- ABBV-1480ABBV-1480 / PD-1, VEGF / Biologic bispecific antibody / OncologySolid TumorsPhase 1
- LutikizumabABT-981 / IL-1a/1b / Biologic / ImmunologyHidradenitis SuppurativaPhase 3
- GemibotulinumtoxinAGemibotA / SNAP-25 / Biologic / NeuroscienceVentral Hernia RepairPhase 2
32assets
- camizestrant + CDK4/6ingSERD + CDK4/6i / small molecule combination / Oncology1L HR+ HER2- ESR1m advanced breast cancerPhase IIISubmitted
- camizestrant + palbociclibngSERD + CDK4/6i / small molecule combination / Oncology1L HR+ HER2- advanced breast cancerPhase III
- camizestrant +/- abemaciclibngSERD + CDK4/6i / small molecule combination / Oncologyadjuvant HR+ HER2- early breast cancerPhase III
- camizestrantngSERD / small molecule / Oncologyadjuvant switch HR+ HER2- early breast cancerPhase III
- AZD0120AZD0120 / CD19/BCMA CAR-T / cell therapy / Oncology/Rare Disease/Respiratory & Immunologyrelapsed/refractory multiple myelomaPhase IIINew to Phase IIIamyloid light-chain amyloidosisPhase Isystemic lupus erythematosusPhase I
- puxitatug samrotecanB7-H4 TOP1i ADC / antibody-drug conjugate / Oncology2-3L B7-H4+ endometrial cancerPhase III
- rilvegostomigPD-1/TIGIT bispecific mAb / bispecific antibody / OncologyHCC; biliary tract; NSCLC; gastric cancerPhase III
- saruparibPARP1 inhibitor / small molecule / Oncologyprostate cancer; breast cancerPhase III
- sonesitatug vedotinCLDN18.2 MMAE ADC / antibody-drug conjugate / OncologyCLDN18.2+ gastric cancerPhase III
- surovatamigAZD0486 / CD19/CD3 T-cell engager / bispecific antibody / Oncology/Respiratory & Immunology1L elderly DLBCL; follicular lymphomaPhase IIIB-cell driven autoimmune diseasePhase I
- torvutatug samrotecanAZD5335 / anti-FRα TOP1i ADC / antibody-drug conjugate / Oncologyovarian cancerPhase III
- volrustomigPD-1/CTLA-4 bispecific mAb / bispecific antibody / Oncologycervical cancer; HNSCC; NSCLC; mesotheliomaPhase IIIcolorectal cancer; NSCLC; other solid tumoursPhase II
- balcinrenone/dapagliflozinMR antagonist/modulator + SGLT2 inhibitor / small molecule combination / Cardiovascular, Renal and Metabolismheart failure with CKDPhase IIICKDPhase II
- baxdrostataldosterone synthase inhibitor / small molecule / Cardiovascular, Renal and MetabolismhypertensionPhase IIIAcceptedprimary aldosteronismPhase III
- baxdrostat/dapagliflozinaldosterone synthase inhibitor + SGLT2 inhibitor / small molecule combination / Cardiovascular, Renal and MetabolismCKD; prevention of heart failurePhase III
- laroprovstatPCSK9 inhibitor / small molecule / Cardiovascular, Renal and MetabolismdyslipidaemiaPhase III
- WainuaWainua / ligand-conjugated antisense / antisense oligonucleotide / Cardiovascular, Renal and MetabolismATTRv-PNPhase IIILaunchedATTR CMLCM
- zibotentan/dapagliflozinendothelin A receptor antagonist/SGLT2 inhibitor / small molecule combination / Cardiovascular, Renal and MetabolismCKD with high proteinuriaPhase III
- elecoglipronoral GLP-1 receptor agonist / small molecule / Cardiovascular, Renal and MetabolismT2D/chronic weight managementPhase II
- AZD6234AZD6234 / peptide / peptide / Cardiovascular, Renal and Metabolismchronic weight management in overweight or obesityPhase II
- tozorakimabIL-33 mAb / monoclonal antibody / Respiratory & ImmunologyCOPDPhase IIIsevere viral lower respiratory tract diseasePhase IIIasthmaPhase II
- TezspireTezspire / TSLP mAb / monoclonal antibody / Respiratory & Immunologysevere uncontrolled asthmaPhase IIILaunchedChinaeosinophilic esophagitis; COPD; nasal polypsLCM
- SaphneloSaphnelo / type I IFN receptor mAb / monoclonal antibody / Respiratory & Immunologysystemic lupus erythematosusPhase IIILaunchedChinacutaneous lupus erythematosus; SLE SCLCM
- anselamimabfibril-reactive mAb / monoclonal antibody / Rare Diseaseamyloid light-chain amyloidosisPhase IIISubmitted
- cliramitugtransthyretin depleter / large molecule / Rare Diseasetransthyretin amyloid cardiomyopathyPhase III
- efzimfotase alfanext generation TNSALP ERT / enzyme replacement therapy / Rare DiseasehypophosphatasiaPhase III
- eneboparatideparathyroid hormone receptor 1 / large molecule / Rare DiseasehypoparathyroidismPhase III
- gefurulimabanti-C5 dual-binding nanobody / nanobody / Rare Diseasegeneralised myasthenia gravisPhase IIISubmitted
- KavigaleKavigale / SARS-CoV-2 LAAB / long-acting antibody / Infectious Diseaseprevention of COVID-19Phase IIILaunched
- AZD0292AZD0292 / pseudomonas Psl-PcrV bispecific mAb / bispecific antibody / Infectious DiseasebronchiectasisPhase II
- AZD5148AZD5148 / anti-Clostridioides difficile TcdB mAb / monoclonal antibody / Infectious Diseasereduction of C. diff recurrencePhase II
- AZD7760AZD7760 / S. aureus virulence factors / mAb combination / Infectious Diseaseprevention of Staph aureus infectionPhase II
30assets
- erenumab-aooeAIMOVIG / CGRP receptor / Monoclonal antibody / NeurosciencePediatric migraineInvestigational
- blinatumomabBLINCYTO / CD19 x CD3 / BiTE molecule / Hematology/Oncology; InflammationNewly diagnosed B-cell precursor acute lymphoblastic leukemiaInvestigationalRelapsed/refractory acute lymphoblastic leukemia, subcutaneous administrationPhase 2InvestigationalSystemic lupus erythematosus with and without nephritisInvestigational
- dazodalibepCD40L / Fusion protein / Rare DiseaseSjögren’s diseaseInvestigational
- romosozumab-aqqgEVENITY / Sclerostin / Monoclonal antibody / BonePediatric osteogenesis imperfectaInvestigational
- tarlatamab-dlleIMDELLTRA / DLL3 x CD3 / BiTE molecule / Hematology/OncologySmall 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 DiseaseUncontrolled gout, subcutaneous administration with methotrexateInvestigational
- sotorasibLUMAKRAS / KRAS G12C / Small molecule / Hematology/OncologyKRAS 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; DiabetesObesityInvestigationalCardiovascular risk reduction in ASCVD with obesity or overweightInvestigationalHeart failure events and cardiovascular risk reduction in HFpEF/HFmrEF with obesityInvestigational
- romiplostimNPLATE / Thrombopoietin receptor / Peptibody / Hematology/OncologyChemotherapy-induced thrombocytopeniaInvestigational
- olpasiranAMG 890 / Lp(a) / siRNA / CardiometabolicAtherosclerotic cardiovascular diseaseInvestigational
- apremilastOTEZLA / PDE4 / Small molecule / InflammationJuvenile psoriatic arthritisInvestigationalPediatric Behcet’s diseaseInvestigational
- etelcalcetidePARSABIV / Calcium-sensing receptor / Peptide / NephrologyPediatric secondary hyperparathyroidism in CKD receiving hemodialysisInvestigational
- evolocumabREPATHA / PCSK9 / Monoclonal antibody / CardiometabolicHypercholesterolemia in high cardiovascular risk without prior MI or strokeInvestigational
- avacopanTAVNEOS / C5aR1 / Small molecule / Rare DiseasePediatric ANCA-associated vasculitisInvestigational
- teprotumumab-trbwTEPEZZA / IGF-1R / Monoclonal antibody / Rare DiseaseThyroid eye disease, subcutaneous administrationInvestigational
- tezepelumab-ekkoTEZSPIRE / TSLP / Monoclonal antibody / InflammationChronic obstructive pulmonary diseaseInvestigationalEosinophilic esophagitisInvestigational
- xaluritamigAMG 509 / STEAP1 x CD3 / XmAb bispecific T-cell engager antibody / Hematology/OncologyProstate cancerInvestigationalEwing sarcomaInvestigational
- ABP 206ABP 206 / PD-1 / Monoclonal antibody biosimilar / Hematology/OncologyInvestigational biosimilar to OPDIVO (nivolumab)Investigational biosimilar
- ABP 234ABP 234 / PD-1 / Monoclonal antibody biosimilar / Hematology/OncologyInvestigational biosimilar to KEYTRUDA (pembrolizumab)Investigational biosimilar
- ABP 692ABP 692 / CD20 / Monoclonal antibody biosimilar / InflammationInvestigational biosimilar to OCREVUS (ocrelizumab)Investigational biosimilar
- daxdilimabILT7 / Monoclonal antibody / Rare DiseaseDermatomyositis and anti-synthetase inflammatory myositisInvestigationalDiscoid lupus erythematosusInvestigational
- inebilizumabUPLIZNA / CD19 / Monoclonal antibody / InflammationSystemic lupus erythematosus with nephritisInvestigational
- carfilzomibKYPROLIS / Proteasome / Small molecule / Hematology/OncologyPediatric acute lymphoblastic leukemiaInvestigational
- AMG 104AMG 104 / TSLP / Monoclonal antibody Fab / InflammationAsthmaInvestigational
- AMG 732AMG 732 / IGF-1R / Monoclonal antibody / Rare DiseaseThyroid eye diseaseInvestigational
- AMG 355AMG 355 / CCR8 / Monoclonal antibody / Hematology/OncologyAdvanced solid tumor malignanciesInvestigational
- AMG 410AMG 410 / Small molecule / Hematology/OncologySolid tumorsInvestigational
- AMG 436AMG 436 / Small molecule / Hematology/OncologyAdvanced solid tumor malignanciesInvestigational
- AMG 513AMG 513 / CardiometabolicObesityInvestigational
- AMG 691AMG 691 / Monoclonal antibody / InflammationAsthmaInvestigational
30assets
- Glucose sensitive insulinNN1845 / Analogue / DiabetesType 1 and 2 diabetesPhase 1
- Ventus NLRP3iNN6022 / NLRP3 / Inhibitor / DiabetesCVDPhase 1
- siRNA GalXC-GYS2siRNA / DiabetessiRNA GalXC-GYS2Phase 1
- CNPCNP / Analogue / DiabetesHeart failurePhase 1
- Amylin 355Analogue / ObesityObesityPhase 1
- Amylin 1213Analogue / ObesityObesityPhase 1
- SLC25A5SLC25A5 / siRNA / ObesityMASHPhase 1
- Inno8Antibody fragment / Rare Blood DisordersHaemophiliaPhase 1
- Subcutaneous ZenagamtideGLP-1 / amylin / Co-agonist / Diabetes / ObesitySubcutaneous ZenagamtidePhase 2ObesityPhase 2
- Monlunabant in DKDCB1 receptor / Small molecule / DiabetesDiabetic Kidney Disease (DKD)Phase 2
- Oral ZenagamtideGLP-1 / amylin / Co-agonist / Diabetes / ObesityDiabetesPhase 2ObesityPhase 2
- CDR132LmiR-132 / Antisense oligonucleotide / DiabetesHeart failure with reduced ejection fraction (HFrEF)Phase 2
- UBT251 (GGG tri-agonist)ObesityObesityPhase 2
- TripleGLP1 / GIP / amylin / Tri-agonist / ObesityObesityPhase 2
- MonlunabantINV-202 / CB1 receptor / Small molecule / ObesityObesityPhase 2
- NDecEPI01 / Rare Blood DisordersSickle cell disease / An oral combination of decitabine and tetrahydrouridine. Project is developed in collaboration with EpiDestiny.Phase 2
- EtavopivatRare Blood DisordersThalassemiaPhase 2
- CagriSema in T2DNN9388 / Analogue / DiabetesType 2 diabetesPhase 3
- Ziltivekimab ASCVDNN6018 / DiabetesAtherosclerotic cardiovascular disease (ASCVD) with chronic kidney disease (CKD) and inflammationPhase 3
- CoramitugPRX004 / DiabetesATTR CardiomyopathyPhase 3
- Ziltivekimabin HFpEFDiabetesHeart failure with preserved ejection fraction (HFpEF)Phase 3
- Ziltivekimab AMIDiabetesAcute Myocardial infarction (AMI)Phase 3
- CagrilintideAnalogue / ObesityObesityPhase 3
- Efruxifermin in MASHFGF21 / Analogue / ObesityMASHPhase 3
- Etavopivat (Sickle Cell Disease)Small molecule / Rare Blood DisordersSickle Cell DiseasePhase 3
- IcosemaNN1535 / Analogue / DiabetesType 2 diabetes / A combination of GLP-1 analogue semaglutide and insulin icodec intended for once-weekly treatment.Filed
- Insulin icodecNN1436 / Analogue / DiabetesDiabetesFiled
- CagriSemaNN9898 / Analogue / ObesityObesityFiled
- Semaglutide 7.2 mgAnalogue / ObesityObesityFiled
- DenecimigMim8 / Inhibitor / Rare Blood DisordersHaemophilia 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 diseasesRSV adults (50-59 years of age at increased risk)Phase III/Registration
- gepotidacinGSK2140944 / Bacterial topoisomerase inhibitor / Small molecule / Infectious diseasesUrogenital gonorrhoeaPhase III/Registration
- bepirovirsenGSK3228836 / HBsAg / Antisense oligonucleotide / Infectious diseasesChronic HBV infectionPhase III
- BexseroBexsero / MenB / Recombinant protein, OMV vaccine / Infectious diseasesMeningitis B (infants US)Phase III/RegistrationUS
- MenABCWY vaccineGSK3536819 / Meningococcal ABCWY / Recombinant protein, OMV, conjugated vaccine / Infectious diseasesMeningococcal diseasePhase III/Registration
- Nucala (mepolizumab)Nucala / IL-5 / Monoclonal antibody / Respiratory/ImmunologyCOPD with eosinophilic phenotypePhase III/Registration
- depemokimabGSK3511294 / IL-5 / Monoclonal antibody / Respiratory/ImmunologyAsthma with type 2 inflammationPhase III/RegistrationChronic rhinosinusitis with nasal polypsPhase III/RegistrationEGPAPhase III
- camlipixantGSK4523199 / P2X3 / Small molecule antagonist / Respiratory/ImmunologyRefractory chronic coughPhase III
- linerixibatGSK2330672 / IBAT / Small molecule / Respiratory/ImmunologyCholestatic pruritus in primary biliary cholangitisPhase III
- Blenrep (belantamab mafodotin)GSK2857916 / Blenrep / BCMA / Antibody-drug conjugate / OncologyRelapsed/refractory multiple myelomaPhase III/Registration
- Jemperli (dostarlimab)Jemperli / PD-1 / Monoclonal antibody / OncologyEndometrial cancerPhase III/RegistrationRectal cancer (dMMR)Phase IINon-small cell lung cancerPhase III
- Zejula (niraparib)Zejula / PARP / Small molecule / OncologyOvarian cancerPhase III/Registration
- belrestotugGSK4428859 / TIGIT / Monoclonal antibody / OncologyNSCLC and other solid tumorsPhase III
- GSK5764227GSK5764227 / B7-H3 / Antibody-drug conjugate / OncologyExtensive-stage small-cell lung cancerPhase IIIAdvanced GI solid tumorsPhase I/II
- cobolimabGSK4069889 / TIM-3 / Monoclonal antibody / OncologyNon-small cell lung cancerPhase II/III
- GSK4532990GSK4532990 / HSD17B13 / RNA interference / Opportunity drivenNASH/MASHPhase II
- GSK4172239GSK4172239 / DNMT1 / Small molecule inhibitor / Opportunity drivenSickle cell diseasePhase I
- GSK3882347GSK3882347 / FimH / Small molecule antagonist / Infectious diseasesUncomplicated UTIPhase I
- GSK3923868GSK3923868 / PI4K beta / Small molecule inhibitor / Infectious diseasesRhinovirus diseasePhase I
- GSK3965193GSK3965193 / PAPD5/PAPD7 / Small molecule inhibitor / Infectious diseasesChronic HBV infectionPhase I
- GSK2556286GSK2556286 / Mtb cholesterol-dependent / Small molecule inhibitor / Infectious diseasesTuberculosisPhase I
- GSK3772701GSK3772701 / P. falciparum whole cell / Small molecule inhibitor / Infectious diseasesMalariaPhase I
- GSK4024484GSK4024484 / P. falciparum whole cell / Small molecule inhibitor / Infectious diseasesMalariaPhase I
- GSK3536867GSK3536867 / Salmonella typhoid + paratyphoid A / Bivalent conjugate vaccine / Infectious diseasesSalmonella (typhoid + paratyphoid A)Phase I
- AGN-151586 / progranulin program (latozinemab)AL001 / Progranulin/sortilin / Monoclonal antibody / Opportunity drivenFrontotemporal dementia with progranulin gene mutationPhase III
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- ZanubrutinibBTK / Inhibitor / Hematologic Malignancies / Immunology & InflammationTreatment-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 MalignanciesTreatment-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 CancersFirst-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 & InflammationRelapsed/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 MalignanciesRelapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (subcutaneous formulation)Phase 2
- TarlatamabDLL3 x CD3 / BiTE® [Bispecific T-cell Engager] / Lung CancersFirst-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 CancersFirst-Line HER2 Positive Gastroesophageal AdenocarcinomaPhase 3Second-Line HER2 Positive Biliary Tract CancerApprovedFirst-Line HER2 positive Biliary Tract CancerPhase 3
- BG-68501CDK2 / Inhibitor / Breast/Gynecologic CancersBreast Cancer and Solid TumorsPhase 1
- BGB-21447BCL2 / Inhibitor / Breast/Gynecologic CancersHormone Receptor Positive Breast CancerPhase 1
- BGB-43395CDK4 / Inhibitor / Breast/Gynecologic CancersBreast Cancer and Solid TumorsPhase 1
- BGB-B455Claudin 6 x CD3 Bispecific / Antibody / Breast/Gynecologic CancersGynecologic and Solid TumorsPhase 1
- BG-C9074B7-H4 Antibody Drug Conjugate / Breast/Gynecologic CancersBreast Cancer and Solid TumorsPhase 1
- BG-75202KAT6A/B / Inhibitor / Breast/Gynecologic CancersBreast CancerPhase 1
- BG-75098CDK2 Chimeric Degradation Activation Compound [CDAC] / Breast/Gynecologic CancersBreast CancerPhase 1
- BGB-B2033GPC3 x 4-1BB Bispecific / Antibody / Gastrointestinal CancersSolid TumorsPhase 1
- BG-C137FGFR2b Antibody Drug Conjugate / Gastrointestinal CancersGastrointestinal CancersPhase 1
- BGB-53038Pan-KRAS / Inhibitor / Gastrointestinal CancersLung Cancers and GI CancersPhase 1
- BG-C477CEA Antibody Drug Conjugate / Gastrointestinal CancersLung Cancers and GI CancersPhase 1
- BG-C0902EGFR x MET x MET Antibody Drug Conjugate / Lung CancersLung Cancers and GI CancersPhase 1
- BG-T187EGFR x MET x MET Trispecific / Antibody / Lung CancersLung Cancers and GI CancersPhase 1
- BGB-58067MTA Cooperative PRMT5 / Inhibitor / Lung CancersLung Cancers and GI CancersPhase 1
- BG-89894MAT2A / Inhibitor / Lung CancersLung Cancers and Solid TumorsPhase 1
- BG-60366EGFR Chimeric Degradation Activation Compound [CDAC] / Lung CancersLung CancersPhase 1
- BGB-26808HPK1 / Inhibitor / Gastrointestinal CancersSolid Tumors/Pan- TumorPhase 1
- XaluritamigSTEAP1 x CD3 Bispecific T-cell Engager (XmAb®) / Other CancersMetastatic Castration-Resistant Prostate CancerPhase 1
- BGB-45035IRAK4 Chimeric Degradation Activation Compound [CDAC] / Immunology & InflammationRheumatoid ArthritisPhase 2Skin DiseasesPhase 1
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- NucresiranRNAi therapeutic / Transthyretin Amyloidosis (TTR)ATTR Amyloidosis with CardiomyopathyPhase 3hATTR Amyloidosis with PolyneuropathyPhase 3
- ZilebesiranRNAi therapeutic / CardiovascularHypertensionPhase 3REVERSIR HypertensionPhase 1
- RapirosiranALN-HSD / HSD / RNAi therapeutic / MetabolicMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 2
- ALN-ANG3ALN-ANG3 / ANG3 / RNAi therapeutic / MetabolicDiabetic Kidney DiseasePhase 2
- ALN-4324ALN-4324 / GRB14 / RNAi therapeutic / MetabolicType 2 Diabetes MellitusPhase 1
- ALN-2232ALN-2232 / ACVR1C / RNAi therapeutic / MetabolicObesity & Weight ManagementPhase 1
- ALN-PNPALN-PNP / PNP / RNAi therapeutic / MetabolicNon-Alcoholic Fatty Liver Disease (NAFLD)Phase 1
- ALN-APOC3ALN-APOC3 / APOC3 / RNAi therapeutic / MetabolicDyslipidemiaPhase 1
- ALN-CIDEBALN-CIDEB / CIDEB / RNAi therapeutic / MetabolicMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 1
- Cemdisiran(+/- Pozelimab)RNAi therapeutic / Neuroscience/OtherMyasthenia GravisPhase 3Geographic AtrophyPhase 3
- MivelsiranALN-APP / APP / RNAi therapeutic / NeuroscienceCerebral Amyloid Angiopathy (CAA)Phase 2Alzheimer’s DiseasePhase 1
- ALN-HTT02ALN-HTT02 / HTT / RNAi therapeutic / NeuroscienceHuntington’s DiseasePhase 1
- ALN-5288ALN-5288 / MAPT / RNAi therapeutic / NeuroscienceAlzheimer’s DiseasePhase 1
- ALN-SODALN-SOD / SOD1 / RNAi therapeutic / NeuroscienceSOD1 Amyotrophic Lateral Sclerosis (ALS)Phase 1
- ALN-SNCAALN-SNCA / SNCA / RNAi therapeutic / NeuroscienceParkinson’s DiseasePhase 1
- Cemdisiran (+Pozelimab)RNAi therapeutic / HematologyParoxysmal Nocturnal HemoglobinuriaPhase 3
- ALN-6400ALN-6400 / PLG / RNAi therapeutic / HematologyBleeding DisordersPhase 2
- AG-236ALN-TMP / TMP / RNAi therapeutic / HematologyPolycythemia VeraPhase 1
- ALN-CFBALN-CFB / CFB / RNAi therapeutic / HematologyParoxysmal Nocturnal HemoglobinuriaPhase 1
- Elebsiran (+Tobevibart)RNAi therapeutic / OtherHepatitis D Virus InfectionPhase 2
- ALN-BCATALN-BCAT / BCAT / RNAi therapeutic / OtherHepatocellular CarcinomaPhase 1
- ALN-4285ALN-4285 / RNAi therapeutic / OtherHealthy VolunteersPhase 1
- ALN-4915ALN-4915 / RNAi therapeutic / OtherHealthy VolunteersPhase 1
- ALN-F1202ALN-F1202 / RNAi therapeutic / OtherHealthy VolunteersPhase 1
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- PumitamigPD-L1/VEGF-A / immunomodulator / OncologyMultiple solid tumors; NSCLC; SCLC; HCC; CRC; ES-SCLC; EGFRm NSCLC; glioblastoma; MPM; NEN; TNBC; PDACclinical
- BNT325/DB-1305BNT325/DB-1305 / targeted therapy / OncologyMultiple solid tumorsclinical
- BNT324/DB-1311BNT324/DB-1311 / targeted therapy / OncologyMultiple solid tumorsclinical
- BNT321BNT321 / targeted therapy / OncologyMultiple solid tumors; HCC combinationclinical
- Autogene cevumeranmRNA cancer immunotherapy / OncologyAdjuvant CRC; adjuvant PDACclinical
- BNT116BNT116 / mRNA cancer immunotherapy / OncologyAdvanced NSCLC; 1L advanced NSCLCclinical
- BNT113BNT113 / HPV16 / mRNA cancer immunotherapy / Oncology1L HPV16+ HNSCCclinical
- GotistobartCTLA-4 / immunomodulator / OncologyMetastatic NSCLC; metastatic CRPC; PROC; multiple solid tumorsclinical
- BNT211BNT211 / cell therapy / OncologyMultiple solid tumorsclinical
- BNT314/GEN1059BNT314/GEN1059 / immunomodulator / OncologyMultiple solid tumors; metastatic CRC combinationclinical
- BNT329BNT329 / immunomodulator / OncologyMultiple solid tumorsclinical
- BNT326/YL202BNT326/YL202 / targeted therapy / OncologyMultiple solid tumors; NSCLC combination; advanced/metastatic breast cancerclinical
- BNT317BNT317 / immunomodulator / OncologyMultiple solid tumorsclinical
- Trastuzumab pamirtecanHER2 / targeted therapy / OncologyMetastatic breast cancer; advanced/metastatic breast cancer combination; 2L endometrial cancer; multiple solid tumorsclinical
- BNT162 + BNT161BNT162 + BNT161 / mRNA vaccine combination / Infectious diseasesCOVID-19-influenza combinationclinical
- BNT163BNT163 / HSV / mRNA vaccine / Infectious diseasesHSVclinical
- BNT166BNT166 / Mpox / mRNA vaccine / Infectious diseasesMpoxclinical
- BNT162BNT162 / COMIRNATY / SARS-CoV-2 / mRNA vaccine / Infectious diseasesCOVID-19Commercialmarketed
- BNT351BNT351 / HIV / antibody mRNA / Infectious diseasesHIVclinical
- BNT164BNT164 / Tuberculosis / mRNA vaccine / Infectious diseasesTuberculosisclinical
- BNT165BNT165 / Malaria / mRNA vaccine / Infectious diseasesMalariaclinical
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- LenacapavirHIV capsid inhibitor / Long-acting injectable / VirologyHIV Prevention (PrEP, PURPOSE 365)Phase 3HIV Treatment (in combinations)Phase 3HIV Treatment (with bNAbs)Phase 2
- Bictegravir/lenacapavir oral combinationINSTI + capsid inhibitor / Oral / VirologyHIV Treatment (virologically suppressed, ARTISTRY-1 & ARTISTRY-2)Phase 3
- Islatravir/lenacapavir oral combinationLong-acting oral / VirologyHIV Treatment (virologically suppressed, ISLEND-1 & ISLEND-2)Phase 3
- Lenacapavir + teropavimab + zinlirvimabCapsid + bNAbs / Long-acting injectable / VirologyHIV Treatment (virologically suppressed)Phase 2
- GS-1720/GS-4182GS-1720/GS-4182 / INSTI/capsid inhibitor / Long-acting oral / VirologyHIV Treatment (WONDERS-1 & -2)Phase 2
- Teropavimab + zinlirvimabbNAbs / Broadly neutralizing antibodies / VirologyHIV CurePhase 2
- VesatolimodVirologyHIV Cure (FRESH)Phase 2
- SelgantolimodVirologyHBV CurePhase 2
- HepcludexHepcludex / VirologyChronic HDV with compensated liver disease (MYR301)FiledBLA pending (US); Approved (EU)USEU
- EdecesertibInflammationLupus (COSMIC)Phase 2
- Tilpisertib fosmecarbilInflammationInflammatory bowel disease (PALEKONA)Phase 2
- α4β7 inhibitorα4β7 / InflammationInflammatory Bowel Disease (SWIFT)Phase 2
- Sacituzumab govitecan-hziyAntibody-drug conjugate / Oncology1L 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 / Oncology2L+ high-risk follicular lymphoma (ZUMA-22)Phase 31L high-risk large B-cell lymphoma (ZUMA-23)Phase 3
- Brexucabtagene autoleucelCD19 CAR / Cell therapy / OncologyPediatric acute lymphocytic leukemia/non-Hodgkin lymphoma (ZUMA-4)Phase 2
- Anitocabtagene autoleucelCell therapy / Oncology2-4L relapsed/refractory multiple myeloma (iMMagine-3)Phase 34L+ relapsed/refractory multiple myeloma (iMMagine-1)FiledBLA pendingUS
- KITE-363KITE-363 / CD19/CD20 bicistronic / Cell therapy / OncologyRelapsed/refractory diffuse large B-cell lymphomaPhase 1RMAT designationRheumatology (autoimmune)Phase 1
- DenikitugGS-1811 / OncologyAdvanced cancersPhase 1
- Domvanalimab + zimberelimabAnti-TIGIT + anti-PD1 / Combination therapy / Oncology1L metastatic non-small cell lung cancer (with chemo, STAR-121)Phase 31L head and neck squamous cell carcinoma (VELOCITY-HNSCC)Phase 2
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- ZilganersenION373 / GFAP / RNA-Targeted Therapeutic / NeurologyAlexander DiseasePhase 1/3ActiveGlobal
- ION582ION582 / UBE3A-ATS / RNA-Targeted Therapeutic / NeurologyAngelman SyndromeClinicalActive
- ION269ION269 / APP / RNA-Targeted Therapeutic / NeurologyAlzheimer's DiseaseClinicalActive
- ION717ION717 / PRNP / RNA-Targeted Therapeutic / NeurologyPrion DiseaseClinicalActive
- ION356ION356 / PLP1 / RNA-Targeted Therapeutic / NeurologyPelizaeus-Merzbacher DiseaseClinicalActive
- ION859ION859 / LRRK2 / RNA-Targeted Therapeutic / NeurologyParkinson's DiseaseClinicalActive
- ION464ION464 / SNCA / RNA-Targeted Therapeutic / NeurologyMultiple System AtrophyClinicalActiveParkinson's DiseaseClinicalActive
- ION440ION440 / MECP2 / RNA-Targeted Therapeutic / NeurologyMECP2 Duplication SyndromeClinicalActive
- OlezarsenApoC-III / RNA-Targeted Therapeutic / CardiologySevere HypertriglyceridemiaClinicalActive
- DonidalorsenPKK / RNA-Targeted Therapeutic / Specialty/RareHereditary AngioedemaClinicalActive
- SapablursenTMPRSS6 / RNA-Targeted Therapeutic / Specialty/RarePolycythemia VeraClinicalActive
- UlefnersenFUS / RNA-Targeted Therapeutic / NeurologyAmyotrophic Lateral SclerosisClinicalActive
- TofersenSOD1 / RNA-Targeted Therapeutic / NeurologyAmyotrophic Lateral Sclerosis (Presymptomatic SOD1)ClinicalActive
- IONIS-MAPTRxBIIB080 / TAU / RNA-Targeted Therapeutic / NeurologyAlzheimer's DiseaseClinicalActive
- TominersenIONIS-HTTRx / HTT / RNA-Targeted Therapeutic / NeurologyHuntington's DiseasePhase 2Active
- ION306ION306 / SMN2 / RNA-Targeted Therapeutic / NeurologySpinal Muscular AtrophyClinicalActive
- EplontersenTTR / RNA-Targeted Therapeutic / CardiologyTransthyretin-Mediated Amyloid CardiomyopathyPhase 3Active
- ION904ION904 / Angiotensinogen / RNA-Targeted Therapeutic / CardiologyTreatment-Resistant HypertensionClinicalActive
- BepirovirsenHepatitis B Virus / RNA-Targeted Therapeutic / Other MedicinesHepatitis B Virus InfectionPhase 3Active
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- zolbetuximabIMAB362 / VYLOY / Claudin 18.2 / Antibody / OncologyGastric and gastroesophageal junction adenocarcinoma, combo with pembrolizumab and chemotherapyFiledEuropeJapan
- gilteritinibASP2215 / XOSPATA / FLT3 / Small molecule / OncologyNewly 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) / OncologyCisplatin-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 HealthVasomotor 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 RegulationPompe diseasePhase 1
- ASP2957ASP2957 / MTM1 gene replacement / Gene therapy (AAV-based gene therapy) / Genetic RegulationX-linked myotubular myopathyPhase 1
- setidegrasibKRAS G12D / Small molecule degrader / Targeted Protein DegradationPancreatic ductal adenocarcinomaPhase 3
- ASP7317ASP7317 / Retinal pigment epithelial cells / Cell therapy / OphthalmologyGeographic atrophy secondary to age-related macular degenerationPhase 1
- ASP1002ASP1002 / Claudin 4/CD137 / T-cell engager bispecific antibody / OncologyGastric and gastroesophageal junction adenocarcinoma; pancreatic adenocarcinomaPhase 1
- ASP5834ASP5834 / Pan-KRAS / Small molecule degrader / OncologyCancerPhase 1
- VIR-5500VIR-5500 / PSMA/CD3 / T-cell engager / OncologyProstate cancerPhase 1
- ASP2998ASP2998 / TROP2 / Antibody-drug conjugate (ADC) / OncologyCancerPhase 1
- ASP2138ASP2138 / Claudin 18.2/CD3 / T-cell engager bispecific antibody / OncologyCancerPhase 2
- ASP546CASP546C / Claudin 18.2 / Antibody-drug conjugate (ADC) / OncologyCancerPhase 2Europe
- abiraterone decanoateASP5541/PRL-02 / CYP17 lyase / Small molecule / OncologyProstate cancerPhase 2
- mirabegronYM178 / Beta-3 receptor / Small molecule / UrologyNeurogenic detrusor overactivity in pediatric patients aged 6 months to less than 3 yearsPhase 3Europe
- roxadustatASP1517/FG-4592 / HIF-PH / Small molecule / NephrologyAnemia associated with chronic kidney disease in pediatric patientsPhase 3Europe
- pudexacianinium chlorideASP5354 / Drug-device combination / OtherIntraoperative 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
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- PlozasiranARO-APOC3 / REDEMPLO / APOC3 / RNAi therapeutic / CardiometabolicFamilial chylomicronemia syndromeIn MarketSevere hypertriglyceridemiaPhase 3ActiveHypertriglyceridemiaPhase 3Active
- ZodasiranARO-ANG3 / ANGPTL3 / RNAi therapeutic / CardiometabolicHomozygous familial hypercholesterolemiaPhase 3Recruiting
- OlpasiranAMG 890; ARO-LPA / LPA / RNAi therapeutic / Cardiovascular diseaseElevated lipoprotein(a)/cardiovascular riskPhase 3Recruiting/Active
- GSK4532990ARO-HSD / HSD17B13 / RNAi therapeutic / Liver diseaseMASH/NASHPhase 2Recruiting
- ARO-PNPLA3JNJ-75220795 / PNPLA3 / RNAi therapeutic / Liver diseaseMASH/NASHPhase 1
- ARO-INHBEINHBE/Activin E / RNAi therapeutic / Obesity/metabolic diseaseObesity with or without type 2 diabetesPhase 1
- ARO-ALK7ACVR1C/ALK7 / RNAi therapeutic / Obesity/metabolic diseaseObesity with or without type 2 diabetesPhase 1Recruiting
- ARO-MAPTMAPT/tau / RNAi therapeutic / CNSAlzheimer's diseasePhase 1Recruiting
- ARO-ATXN2ATXN2 / RNAi therapeutic / CNSSpinocerebellar ataxia 2Phase 1
- SRP-1005ARO-HTT / HTT / siRNA therapeutic / CNSHuntington's diseasePhase 1
- ARO-RAGERAGE / RNAi therapeutic / PulmonaryInflammatory pulmonary diseasesPhase 2Active
- ARO-MMP7MMP7 / RNAi therapeutic / PulmonaryIdiopathic pulmonary fibrosisPhase 1
- FazirsiranTAK-999; ARO-AAT / Z-AAT / RNAi therapeutic / Liver diseaseAlpha-1 antitrypsin deficiency-associated liver diseasePhase 3Recruiting
- ARO-HBVJNJ-3989; JNJ-73763989 / HBV gene products / RNAi therapeutic / Infectious diseaseChronic hepatitis BPhase 2
- ARO-DUX4DUX4 / RNAi therapeutic / Muscular diseaseFacioscapulohumeral muscular dystrophyPhase 2
- ARO-DM1DMPK mRNA / RNAi therapeutic / Muscular diseaseMyotonic dystrophy type 1Phase 2Recruiting
- ARO-C3Complement component 3 / RNAi therapeutic / Complement mediated diseaseComplement-mediated renal diseasePhase 1RecruitingC3 glomerulopathyPhase 1RecruitingIgA nephropathyPhase 1Recruiting
- ARO-CFBComplement factor B / RNAi therapeutic / Complement mediated diseaseComplement mediated diseasePhase 1
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- Vabysmofaricimab-svoa / VABYSMO / VEGF/Ang-2 / Bispecific antibody / OphthalmologyNeovascular age-related macular degeneration (nAMD) and Diabetic macular edema (DME)Approvedapproved
- Ojemdatovorafenib / OJEMDA / OncologyOncology (undisclosed approved indication)Approvedapproved
- Miplyffaarimoclomol / MIPLYFFA / Rare Diseases / NeurologyNiemann-Pick disease type C (NPC)ApprovedapprovedUS
- Xaciatoclindamycin phosphate vaginal gel 2% / XACIATO / Small molecule / Gynecology / Infectious DiseaseBacterial VaginosisApprovedapproved
- Ixinitycoagulation factor IX (recombinant) / IXINITY / Factor IX / Recombinant protein / HematologyHemophilia BApprovedapproved
- Dsuviasufentanil sublingual tablet / DSUVIA / Opioid receptor / Small molecule / Pain ManagementPainApprovedapproved
- Dare to Playsildenafil cream / DARE to PLAY / PDE5 / Small molecule / Gynecology / Sexual HealthFemale Sexual Arousal DisorderApprovedapproved
- ersodetugRZ358 / INSR / Antibody / Metabolic DisordersHypoglycemia due to congenital hyperinsulinism (cHI)Phase 3ongoingHypoglycemia due to tumor hyperinsulinismPhase 3ongoing
- RZ402RZ402 / OphthalmologyDiabetic macular edema (DME)Phase 2ongoing
- D-FiFCX-007 / Gene Therapy / Dermatology / Rare DiseasesDystrophic epidermolysis bullosa (DEB)Phase 3ongoing
- seralutinibGB002 / PDGFR, CSF1R, and c-KIT / Inhaled tyrosine kinase inhibitor / Respiratory / CardiovascularPulmonary arterial hypertension (PAH)Phase 3ongoingPulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD)Phase 2ongoing
- cetrelimabJNJ-63723283 / PD-1 / Antibody / Oncology / Infectious DiseaseMuscle-invasive bladder cancer (MIBC)Phase 2ongoingChronic Hepatitis B Virus InfectionPhase 2ongoing
- volixibatLUM-002 / Ileal bile acid transporter (IBAT) / Small molecule / GastroenterologyPrimary sclerosing cholangitis (PSC)Phase 2bongoingPrimary biliary cholangitis (PBC)Phase 2ongoing
- osavampatorNeurology / PsychiatryMajor depressive disorderPhase 3ongoing
- OHB-607OHB-607 / Recombinant human IGF-I/IGFBP-3 / Recombinant protein / Respiratory / NeonatologyPrevention of bronchopulmonary dysplasia in extremely premature infantsPhase 2ongoing
- REC-4881REC-4881 / Allosteric MEK1/2 / Small molecule / OncologyFamilial adenomatous polyposisPhase 2ongoing
- NIS793NIS793 / Antibody / OncologySolid tumorsPhase 3ongoing
- QTORIN rapamycinQTORIN / mTOR / Small molecule / Dermatology / Rare DiseasesCutaneous venous malformationsPhase 3ongoingClinically significant angiokeratomasPhase 2ongoing
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- direclidineM4 / Small Molecule / PsychiatrySchizophreniaPhase 3Bipolar ManiaPhase 2
- NBI-570M1/M4 / Small Molecule / PsychiatrySchizophreniaPhase 2
- osavampatorAMPA / Small Molecule / PsychiatryMajor Depressive DisorderPhase 3
- NBI-986M4 / Small Molecule / NeurologyMovement DisordersPhase 1
- NBI-890VMAT2 / Small Molecule / PsychiatryTardive DyskinesiaPhase 2
- NBI-569M1/M4 / Small Molecule / PsychiatryAlzheimer's PsychosisPhase 1
- NBI-567M1 / Small Molecule / NeurologyAlzheimer's CognitionPhase 1
- NBI-355Nav1.2/1.6 / Small Molecule / NeurologyEpilepsyPhase 1
- NBIP-1435CRF1 / Peptide / EndocrinologyClassic Congenital Adrenal HyperplasiaPhase 1
- NBIP-2118CRF2 / Peptide / EndocrinologyObesityPhase 1
- NBIP-1968TG + NBIP-2118 Combo / Peptide / EndocrinologyObesityPhase 4
- NBIB-223Frataxin / Gene Therapy / NeurologyFriedreich's AtaxiaPhase 4
- NBI-675VMAT2 / Small Molecule / PsychiatryCNS IndicationsPhase 1
- NBI-188CRF1 / Small Molecule / EndocrinologyWomen's HealthPhase 4
- NBIM-1748ImmunologyUndisclosedPhase 4
- NBIM-1008ImmunologyUndisclosedPhase 4
- NBIM-1112ImmunologyUndisclosedPhase 1
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- FianlimabLAG3 / Monoclonal Antibody / Oncology1L metastatic melanoma, adjuvant melanomaPhase 3ActiveGlobal
- CemiplimabLibtayo / PD-1 / Monoclonal Antibody / OncologyCancerPhase 2/3ActiveGlobal
- DavutamigREGN5093 / METxMET / Bispecific Antibody / OncologyCancerPhase 2ActiveGlobal
- ItepekimabIL-33 / Monoclonal Antibody / Immunology & Inflammation / RespiratoryCOPD, CRSwNPPhase 3ActiveGlobal
- LinvoseltamabLynozyfic / BCMAxCD3 / Bispecific Antibody / OncologyMultiple myelomaPhase 2/3ActiveGlobal
- MarlotamigREGN7075 / EGFRxCD28 / Bispecific Antibody / OncologySolid TumorsPhase 2ActiveGlobal
- OdronextamabOrdspono / CD20xCD3 / Bispecific Antibody / OncologyLymphomaPhase 2ActiveGlobal
- UbamatamabREGN4018 / MUC16xCD3 / Bispecific Antibody / OncologyOvarian CancerPhase 2ActiveGlobal
- NezastomigREGN5678 / PSMAxCD28 / Bispecific Antibody / OncologyProstate CancerPhase 2ActiveGlobal
- RapirosiranALN-HSD / HSD17B13 / siRNA / Cardiovascular & MetabolicNASHPhase 2ActiveGlobal
- ALN-ANG3ALN-ANG3 / ANGPTL3 / siRNA / Cardiovascular & MetabolicDyslipidemiaPhase 2ActiveGlobal
- CemdisiranC5 / siRNA / Ophthalmology / ImmunologyGeographic atrophyClinicalActiveGlobal
- PozelimabC5 / Monoclonal Antibody / Ophthalmology / ImmunologyGeographic atrophyClinicalActiveGlobal
- BetV1REGN5713-5715 / BetV1 / Monoclonal Antibody / Immunology & InflammationBirch AllergyClinicalActiveGlobal
- DB-OTOOtarmeni / Otoferlin / Gene Therapy / Neurology / Hearing LossOtoferlin-related hearing lossPhase 1/2ActiveGlobal
- MibavademabUnknown / Biologic / OncologyCancerPhase 2ActiveGlobal
- TrevogrumabREGN1033 / Myostatin / Monoclonal Antibody / MetabolicMuscle weakness/wastingPhase 2ActiveGlobal
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- mezigdomidecereblon E3 ligase / CELMoD; oral targeted protein degrader / Hematologyrelapsed/refractory multiple myelomaPhase IIIactive
- iberdomidecereblon E3 ligase / CELMoD; oral targeted protein degrader / Hematologymultiple myelomaPhase IIIactive
- golcadomidecereblon E3 ligase / CELMoD; oral targeted protein degrader / Hematologyaggressive B-cell lymphomaPhase IIactive
- pumitamigBNT327/BMS-986545 / PD-L1; VEGF-A / bispecific antibody / Oncologysolid tumorsPhase IIIactive
- iza-brenantibody-drug conjugate / Oncologysolid tumorsPhase I/IIactive
- nivolumabOPDIVO / PD-1 / monoclonal antibody / Oncologymultiple oncology indicationsPhase IIIactive LCM
- nivolumab and relatlimab HDPD-1; LAG-3 / antibody combination / Oncologyoncology indicationsPhase IIIactive LCM
- ipilimumabYERVOY / CTLA-4 / monoclonal antibody / Oncologymultiple oncology indicationsPhase IIIactive LCM
- KarXTCOBENFY / muscarinic receptors / small molecule / Neuroscienceneuropsychiatric indicationsPhase IIIactive LCM
- milvexianFactor XIa / small molecule anticoagulant / Cardiovascularthrombotic disordersPhase IIIactive
- BMS-986278BMS-986278 / LPA1 / small molecule antagonist / Fibrosisidiopathic pulmonary fibrosisPhase IIIactive
- BMS-986495BMS-986495 / MTBR tau / antibody / NeuroscienceAlzheimer's diseasePhase IIactive
- imzokitugCCR8 / monoclonal antibody / Oncologysolid tumorsPhase I/IIactive
- obexelimabCD19; FcγRIIb / bifunctional antibody / Immunologyautoimmune diseasesPhase IIIactive
- luspatercept-aamtREBLOZYL / TGF-beta superfamily ligands / recombinant fusion protein / Hematologyanemia indicationsPhase IIIactive LCM
- OncoACP3OncoACP3 / prostate cancer target / radiopharmaceutical therapeutic/diagnostic agent / Oncologyprostate canceractive
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- guselkumabTREMFYA / IL-23 / Monoclonal Antibody / ImmunologyPsoriasisPsoriatic ArthritisCrohn's Disease
- ibrutinibIMBRUVICA / BTK / Small Molecule / OncologyMantle Cell LymphomaOther B-cell malignancies
- icotrokinraJNJ-2113 / IL-23R / Targeted Oral Peptide / ImmunologyPsoriasisPsoriatic Arthritis
- JNJ-4804JNJ-4804 / Co-antibody / Co-antibody Therapy / ImmunologyUlcerative Colitis
- nipocalimabIMAAVY / FcRn / Monoclonal Antibody / Immunology / NeuroscienceGeneralized Myasthenia GravisRheumatoid ArthritisWarm Autoimmune Hemolytic Anemia
- aticaprantKOR antagonist / Small Molecule / NeuroscienceAdjunctive Treatment for Major Depressive Disorder with Anhedonia
- RPGR Gene TherapyRPGR / Gene Therapy / Vision / OphthalmologyRetinitis Pigmentosa
- teclistamabTECVAYLI / BCMA x CD3 / Bispecific Antibody / OncologyMultiple Myeloma
- sCD59JNJ-1887 / sCD59 / Gene Therapy / Vision / OphthalmologyGeographic Atrophy
- gemcitabine intravesical delivery systemTAR-200 / INLEXZO / DNA synthesis / Drug Delivery System / OncologyHigh Risk Non-Muscle Invasive Bladder Cancer
- niraparib / abirateroneAKEEGA / PARP / CYP17 / Small Molecule Combination / OncologyMetastatic Castration-Sensitive Prostate CancerMetastatic Castration-Resistant Prostate Cancer
- daratumumabDARZALEX / CD38 / Monoclonal Antibody / OncologyMultiple Myeloma
- erdafitinibBALVERSA / FGFR / Small Molecule / OncologyUrothelial Carcinoma
- apalutamideERLEADA / Androgen Receptor / Small Molecule / OncologyProstate Cancer
- ciltacabtagene autoleucelCARVYKTI / BCMA / CAR-T Cell Therapy / OncologyMultiple Myeloma
- Filovirus multivalent vaccineVAC69120 / MVA-BN-Filo / Ebola Virus / Vaccine / Infectious DiseasesEbola
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- NiraparibPARP / Inhibitor / OncologyOvarian Cancer (1st line maintenance)ApprovedOvarian Cancer (Platinum sensitive relapsed maintenance)Approved
- Tumor Treating FieldsOncologyGlioblastoma (GBM)ApprovedPancreatic Cancer (1st line)Phase 3 / Pivotal
- RipretinibTKI / OncologyGastrointestinal Stromal Tumors (GIST) (4th line)Approved
- RepotrectinibROS1 / TRK / OncologyROS1+ NSCLCApprovedNTRK+ solid tumorsApproved
- Tisotumab vedotinTF / ADC / OncologyCervical Cancer (2nd line+ r/m)Phase 3 / PivotalCervical Cancer (1st line r/m, combo)Phase 2 / POC
- Zocilurtatug pelitecanZL-1310 / Zoci / DLL3 / ADC / OncologyES-SCLCPhase 3 / PivotalOther NECsPhase 2 / POC
- ZL-6201LRRC15 / ADC / OncologySarcoma & CAF+ Solid TumorPhase 1
- ZL-1311MUC17xCD3 / OncologyGI TumorsPre-clinical
- ZL-1222PD-1/IL-12 / OncologySolid TumorsPre-clinical
- EfgartigimodFcRn / ImmunologyGeneralized Myasthenia Gravis (gMG)ApprovedChronic Inflammatory Demyelinating Polyneuropathy (CIDP)ApprovedMyositisPhase 3 / Pivotal
- PovetaciceptBAFF/APRIL / ImmunologyIgA NephropathyPhase 3 / PivotalPrimary Membranous NephropathyPhase 2 / POC
- ElegrobartIGF-1R / ImmunologyTEDPhase 3 / Pivotal
- ZL-1503IL13/IL31R / ImmunologyMod-to-Sev ADPhase 1
- Xanomeline-TrospiumKarXT / NeuroscienceSchizophreniaApproved
- OmadacyclineInfectious DiseaseAcute Bacterial Skin and Skin Structure Infection (ABSSSI)ApprovedCommunity-Acquired Bacterial Pneumonia (CABP)Approved
- Sulbactam-DurlobactamInfectious DiseaseHABP/VABP caused by Susceptible Isolates of Acinetobacter Baumannii-calcoaceticus ComplexApproved
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- RelugolixTAK-385
- BrigatinibAlunbrig
- IxazomibNinlaro
- FruquintinibFruzaqla
- RusfertideTAK-121
- ElriterceptTAK-226
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- BotensilimabAGEN1181 / Anti-CTLA-4 / Fc-enhanced anti-CTLA-4 antibody / OncologyNon-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 / OncologyMSS metastatic colorectal cancer in combination with botensilimabPhase 3Sites activePD-1 R/R melanoma in combination with botensilimabPhase 2
- AGEN2373AGEN2373 / CD137 / CD137 agonist antibody / OncologySolid tumorsPhase 1PD-1 R/R melanoma with botensilimabPhase 2
- AGEN1571AGEN1571 / Anti-ILT-2 / Antibody / OncologySolid tumorsPhase 1
- AGEN1777AGEN1777 / Anti-TIGIT x CD96 / Bispecific antibody / OncologySolid tumors with balstilimabPhase 1
- AGEN1423AGEN1423 / Anti-CD73 x TGFB / Bispecific antibody / OncologySolid tumorsPhase 1
- INCAGN1876INCAGN1876 / Anti-GITR / Antibody / OncologySolid tumorsPhase 1
- AGEN1949AGEN1949 / OX40 / OX40 agonist antibody / OncologySolid tumorsPhase 1
- MK-4830MK-4830 / ILT4 / Anti-ILT4 antagonist antibody / OncologyNeoadjuvant ovarian cancerPhase 2Ongoing
- INCAGN2390INCAGN2390 / TIM-3 / Anti-TIM-3 antagonist antibody / OncologyMelanoma and SCCHN in combinationIn development
- INCAGN2385INCAGN2385 / LAG-3 / Anti-LAG-3 antagonist antibody / OncologyMelanoma and SCCHN in combinationIn development
- CR6086 + BalstilimabCR6086; AGEN2034 / EP4 + PD-1 / EP4 antagonist plus PD-1 antagonist / OncologypMMR-MSS colorectal cancerPhase 1/2Ongoing
- NLM001 + ZalifrelimabNLM-001 / Hedgehog + CTLA-4 / Hedgehog inhibitor plus CTLA-4 antagonist / OncologyAdvanced pancreatic cancerPhase 1/2Ongoing
- OBT076 + BalstilimabOBT076; AGEN2034 / CD205 + PD-1 / Antibody-drug conjugate plus PD-1 antibody / OncologySolid tumorsOngoing
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- High-concentration, low-volume adalimumabAVT02 / HUMIRA® / Biosimilar / ImmunologyBiosimilar to HUMIRA®ApprovedApprovedU.S.Europe (EEA, UK, Switzerland, Balkans)CanadaAustraliaSaudi ArabiaIsraelEgyptSouth AfricaMoroccoPeruBolivia
- ustekinumabAVT04 / STELARA® / Biosimilar / ImmunologyBiosimilar to STELARA®ApprovedApprovedU.S.JapanCanadaEU/EEA
- denosumabAVT03 / PROLIA®/XGEVA® / Biosimilar / Bone DiseaseBiosimilar to PROLIA®/XGEVA®ApprovedApprovedJapanEU/EEA
- golimumabAVT05 / SIMPONI®/SIMPONI ARIA® / Biosimilar / ImmunologyBiosimilar to SIMPONI®/SIMPONI ARIA®ApprovedApprovedJapanEU/EEAUnited Kingdom
- afliberceptAVT06 / EYLEA® / Biosimilar / OphthalmologyBiosimilar 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
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- 177Lu rosopatamab tetraxetanTLX591-Tx / PSMA / Antibody / Urologic OncologyProstate Cancer
- 177Lu-DOTA-PSMATLX597-Tx / PSMA / Small molecule / Urologic OncologyProstate Cancer
- 177Lu-girentuximabTLX250-Tx / CAIX / Antibody / Urologic OncologyKidney Cancer / ccRCC
- 131I-IPATLX101-Tx / LAT / Small molecule / Neuro-oncologyBrain Cancer / Glioma
- 90Y-besilesomabTLX66-Tx / CD66 / Antibody / HematologyBone-marrow conditioning
- 225Ac-RADmAbTLX592-Tx / PSMA / Antibody / Urologic OncologyProstate Cancer
- 225Ac-girentuximabTLX252-Tx / CAIX / Antibody / Urologic OncologyKidney Cancer
- UndisclosedTLX400-Tx / FAP / Small molecule / Urologic OncologyBladder Cancer
- 211At-APATLX102-Tx / LAT / Small molecule / Neuro-oncologyBrain Cancer
- -olaratumab conjugateTLX300-Tx / PDGFRα / Antibody / Musculoskeletal OncologySoft Tissue Sarcoma
- 153Sm-DOTMPTLX090-Tx / Small molecule / Musculoskeletal OncologyBone metastasesPain palliation
- 68Ga-PSMA-11TLX591-CDx / Illuccix / Gozellix / PSMA / Diagnostic Radiopharmaceutical / OncologyProstate Cancer Imaging
- 18F-floretyrosineTLX101-CDx / Pixclara / Pixlumi / LAT / Diagnostic Radiopharmaceutical / Neuro-oncologyGlioma Imaging
- 89Zr-DFO-girentuximabTLX250-CDx / Zircaix / CAIX / Diagnostic Radiopharmaceutical / OncologyClear Cell Renal Cell Carcinoma (ccRCC) Imaging
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- EfgartigimodVYVGART; VYVGART Hytrulo / FcRn / antibody fragment / AutoimmuneGeneralized Myasthenia Gravis (gMG)CommercialChronic Inflammatory Demyelinating Polyneuropathy (CIDP)CommercialPrimary Immune Thrombocytopenia (ITP)CommercialJapan
- EmpasiprubartC2 / monoclonal antibody / AutoimmuneMultifocal Motor Neuropathy (MMN)RegistrationalDelayed Graft Function (DGF)Proof of ConceptChronic Inflammatory Demyelinating Polyneuropathy (CIDP)Registrational
- AdimanebartMuSK / agonist antibody / NeurologyCongenital Myasthenic Syndromes (CMS)Proof of ConceptSpinal Muscular Atrophy (SMA)Proof of Concept
- ARGX-109ARGX-109 / IL-6 / antibody / UndisclosedUndisclosedPhase 1
- ARGX-121ARGX-121 / IgA / antibody / UndisclosedUndisclosedPhase 1
- ARGX-213ARGX-213 / FcRn / antibody / UndisclosedUndisclosedPhase 1
- ARGX-118ARGX-118 / Galectin-10 / Airway InflammationAirway InflammationPreclinical
- ARGX-125ARGX-125 / bispecific antibody / UndisclosedUndisclosedPreclinical
- TSP-101TSP-101 / Fn14 / UndisclosedUndisclosedPreclinical
- LP0145ARGX-112 / IL-22R / SIMPLE Antibody / DermatologyAtopic Dermatitispartnered
- AGMB-101ARGX-114 / MET / HGF-mimetic agonist antibody
- ABBV-151ARGX-115 / GARP-TGF-beta1 / SIMPLE Antibody / OncologyTumorspartnered
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- CasdatifanAB521 / HIF-2α Inhibitor
- QuemliclustatCD73 Inhibitor
- DomvanalimabAnti-TIGIT Antibody
- ZimberelimabAnti-PD-1 Antibody
- EtrumadenantA2a/A2b Receptor Antagonist
- AB598Anti-CD39 Antibody
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- Taldefgrobep AlfaBHV-2000 / myostatin/activin receptor signaling / recombinant protein; myostatin-activin pathway inhibitor / neuromuscular; metabolicSpinal muscular atrophyPhase 3ObesityPhase 1
- OpakalimBHV-7000 / Kv7.2/Kv7.3 potassium channels / small molecule Kv7 activator / neurology; psychiatryFocal epilepsyPhase 3Generalized epilepsyPhase 3Major depressive disorderPhase 2
- TRPM3 AntagonistBHV-2100 / TRPM3 / oral small molecule antagonist / painPain disordersPhase 2
- TYK2/JAK1 InhibitorBHV-8000 / TYK2/JAK1 / brain-penetrant small molecule inhibitor / neuroinflammationParkinson's diseasePhase 3Prevention of amyloid therapy induced ARIAPhase 1Alzheimer's diseasePhase 1
- IgG DegraderBHV-1300 / IgG / MoDE extracellular protein degrader / immunologyGraves' disease / rheumatoid arthritisPhase 1
- IgG DegraderBHV-1310 / IgG / MoDE extracellular protein degrader / immunologyMyasthenia gravisPreclinical
- Gd-IgA1 TRAP DegraderBHV-1400 / galactose-deficient IgA1 / TRAP extracellular protein degrader / immunology; renalIgA nephropathyPhase 1
- β1AR Autoantibody DegraderBHV-1600 / β1AR autoantibodies / bifunctional MoDE degrader / cardiovascularPeripartum cardiomyopathyPhase 1
- Trop2 ADC +/- PD1BHV-1510 / TROP2 / antibody-drug conjugate / oncologyAdvanced or metastatic epithelial tumorsPhase 1
- FGFR3 ADCBHV-1530 / FGFR3 / antibody-drug conjugate / oncologyUrothelial cancer and other tumorsPhase 1
- CD30 ADCBHV-1500 / CD30 / antibody-drug conjugate / oncologyHodgkin lymphomaPreclinical
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- LecanemabLEQEMBI / Aβ / mAb / NeurologyPreclinical Alzheimer's diseasePhase 3Early Alzheimer's disease, SC-AI initiationRegulatory review; FDA PDUFA May 24, 2026U.S.
- DiranersenBIIB080 / tau / ASO / NeurologyEarly Alzheimer's diseasePhase 2PoC readout reported May 14, 2026
- BIIB091BIIB091 / BTK / peripheral BTK inhibitor / NeurologyMultiple sclerosisPhase 2
- Dapirolizumab pegolCD40L / pegylated antibody fragment / ImmunologySystemic lupus erythematosusPhase 3
- LitifilimabBDCA2 / mAb / ImmunologySystemic lupus erythematosusPhase 3Cutaneous lupus erythematosusPhase 3FDA Breakthrough Therapy DesignationU.S.
- BIIB142BIIB142 / IRAK4 / degrader / ImmunologyAutoimmune diseasePhase 1
- FelzartamabCD38 / mAb / Rare diseaseAntibody-mediated rejectionPhase 3IgA nephropathyPhase 3Primary membranous nephropathyPhase 3
- OmaveloxoloneSKYCLARYS / Nrf2 / activator / Rare diseasePediatric Friedreich ataxiaPhase 3
- SalanersenBIIB115 / SMN / ASO / Rare diseaseSpinal muscular atrophyPhase 3
- ZorevunersenSCN1A / ASO / Rare diseaseDravet syndromePhase 3
- Nusinersen high doseSPINRAZA / SMN2 / splice modulator / Rare diseaseSpinal muscular atrophyApproved in U.S.; under review in E.U.U.S.E.U.
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- tividenofusp alfa-eknmDNL310; ETV:IDS / AVLAYAH / IDS enzyme replacement; TfR TransportVehicle-enabled delivery / Enzyme TransportVehicle biologic / Lysosomal storage disordersHunter 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 disordersSanfilippo 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 diseaseGRN-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 diseaseAlzheimer's diseasePhase 1bCTA approved; study start-up underway
- DNL952ETV:GAA / GAA enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disordersPompe diseasePhase 1IND clinical hold lifted; proceeding to Phase 1US
- BIIB122/DNL151DNL151; BIIB122 / LRRK2 / Small molecule inhibitor / Neurodegenerative diseaseParkinson's diseasePhase 2bLUMA enrollment completed; readout expected 2026GlobalLRRK2-associated Parkinson's diseasePhase 2aBEACON ongoing
- eclitasertibSAR443122/DNL758 / RIPK1 / Small molecule inhibitor / Inflammatory diseaseUlcerative colitisPhase 2Developed by Sanofi; Phase 2 results expected in 1H 2026
- DNL921ATV:Abeta / Amyloid beta / Antibody TransportVehicle therapeutic / Neurodegenerative diseaseAlzheimer's diseaseIND-enablingPhase 1/1b initiation planned
- DNL111ETV:GCase / GCase / Enzyme TransportVehicle biologic / Neurodegenerative disease; lysosomal storage disordersParkinson's diseaseIND-enablingIND-enablingGaucher diseaseIND-enablingIND-enabling
- DNL622ETV:IDUA / IDUA enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disordersMPS IIND-enablingIND-enabling
- DNL422OTV:SNCA / SNCA / alpha-synuclein / Oligonucleotide TransportVehicle therapeutic / Neurodegenerative diseaseParkinson's diseaseIND-enablingIND-enabling
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- INCA033989INCA033989 / mutCALR / HematologyCALR-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 / Oncology1L 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 / Oncology1L microsatellite-stable colorectal cancer (MSS CRC)Phase 3Ongoing1L MSS PDAC (combo with SOC)Phase 1Data expected 2H 2026
- INCB123667INCB123667 / CDK2 / OncologyPlatinum-resistant ovarian cancer (PROC)PivotalOngoing1L maintenance ovarian cancer (CCNE1+)Phase 3Initiation planned 2H 2026
- povorcitinibJAK1 / small molecule / Inflammation and AutoimmunityModerate-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 AutoimmunityModerate atopic dermatitisApprovedApproval and launch (ex-US)EUMild-to-moderate hidradenitis suppurativa (HS)Phase 3Ongoing; topline data 4Q 2026
- axatilimabNiktimvo / CSF-1R / Hematology1L chronic GVHD (+ ruxolitinib)PivotalData 2H 20261L chronic GVHD (+ steroids)Data early 20283L+ chronic GVHDApprovedApprovedUS
- INCB160058INCB160058 / JAK2V617F / HematologyJAK2 V617F-mutated myeloproliferative neoplasms (MPNs)Phase 1Data 2H 2026 (ASD formulation)
- tafasitamabMonjuvi / CD19 / Hematology1L diffuse large B-cell lymphoma (DLBCL)Phase 3sBLA submission 1H 2026USRelapsed/refractory DLBCLApprovedApprovedUSEUJapan
- ruxolitinib XRJakafi XR / JAK1/JAK2 / HematologyMyelofibrosis, polycythemia vera, chronic GVHDRegistrationFDA resubmissionUS
- retifanlimabZynyz / PD-1 / OncologySquamous cell carcinoma of the anal canal (SCAC)ApprovedApproved (EC approval noted)USEUMerkel cell carcinomaApprovedApprovedUSEUJapan
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- B-VECB-VEC / VYJUVEK / Type VII collagen (COL7A1) / Gene Therapy (HSV-1 vector) / DermatologyDystrophic Epidermolysis Bullosa (DEB)CommercialApprovedGlobal
- KB105KB105 / Transglutaminase 1 (TGM1) / Gene Therapy / DermatologyTGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)Phase 1/2ActiveGlobal
- KB104KB104 / Serine Peptidase Inhibitor Kazal Type 5 (SPINK5) / Gene Therapy / DermatologyNetherton SyndromePreclinicalActiveGlobal
- KB407KB407 / Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) / Gene Therapy / RespiratoryCystic FibrosisPhase 1ActiveGlobal
- KB408KB408 / Alpha-1 antitrypsin / Gene Therapy / RespiratoryAlpha-1 Antitrypsin Deficiency (AATD)PreclinicalActiveGlobal
- KB707KB707 / IL-12 and IL-2 / Gene Therapy (HSV-1 vector) / OncologyLocally advanced or metastatic solid tumor malignanciesPhase 1ActiveGlobal
- KB301KB301 / Type III collagen / Gene Therapy / AestheticsAesthetic Skin ConditionsPhase 1/2ActiveGlobal
- KB302KB302 / Type I collagen / Gene Therapy / AestheticsAesthetic Skin ConditionsPreclinicalActiveGlobal
- KB303KB303 / Elastin / Gene Therapy / AestheticsAesthetic Skin ConditionsPreclinicalActiveGlobal
- KB304KB304 / Type III collagen and Elastin / Gene Therapy / AestheticsAesthetic Skin ConditionsPreclinicalActiveGlobal
- KB305KB305 / Type IV collagen / Gene Therapy / AestheticsAesthetic Skin ConditionsPreclinicalActiveGlobal
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- amlitelimabOX40L / Monoclonal antibody / ImmunologyAtopic dermatitisPhase 3ActiveGlobal
- tolebrutinibBTK / Small molecule / NeurologyMultiple sclerosisRegistrationActiveGlobalUS
- lunsekimigBiologic / Immunology and InflammationAsthma / Respiratory diseasesPhase 2ActiveGlobal
- rilzabrutinibBTK / Small molecule / Rare Blood Disorders / ImmunologySickle cell diseasePhase 2/3ActiveGlobalIgG4-related diseasePhase 3ActiveGlobalAsthmaPhase 2ActiveGlobal
- venglustatSmall molecule / Rare DiseasesGaucher disease type 3 (GD3)Phase 3ActiveGlobal
- frexalimabCD40L / Monoclonal antibody / ImmunologyMultiple sclerosisPhase 3ActiveGlobal
- SAR441566SAR441566 / TNFR1si / Small molecule / ImmunologyImmunological diseasesPhase 2ActiveGlobal
- duvakitugTL1A / Monoclonal antibody / ImmunologyUlcerative colitisPhase 2ActiveGlobalCrohn's diseasePhase 2ActiveGlobal
- itepekimabIL-33 / Monoclonal antibody / Immunology and InflammationCOPDPhase 3ActiveGlobal
- isatuximabSarclisa / CD38 / Monoclonal antibody / OncologyMultiple myeloma (subcutaneous)RegistrationActiveEU
- acoziboroleSmall molecule / Infectious DiseasesSleeping sicknessRegistrationActiveAfricaEU
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- Ozuriftamab vedotinBA3021 / ROR2 / CAB-ROR2-ADC / OncologyOPSCCPhase 3
- Mecbotamab vedotinBA3011 / AXL / CAB-AXL-ADC / OncologySarcoma (soft tissue and bone)Phase 2mKRAS NSCLCPhase 2
- EvalstotugBA3071 / CTLA-4 / CAB-CTLA-4 antibody / OncologyUnresectable and/or metastatic cutaneous melanomaPhase 2
- BA3182BA3182 / EpCAM x CD3 / Dual CAB T cell engager / OncologyAdenocarcinomaPhase 1ongoing
- BA3361BA3361 / Nectin-4 / CAB-Nectin4-ADC / OncologyMultiple tumor typesPreclinicalIND-approved
- BA3151BA3151 / B7-H4 / CAB-B7H4-ADC / OncologyBreast cancerPreclinical
- BA3142BA3142 / B7-H3 x CD3 / Dual CAB TCE / OncologyMultiple tumor typesPreclinicalIND-ready
- BA3311BA3311 / EGFR x CD3 / TCE / OncologyMultiple tumor typesPreclinical
- BA3241BA3241 / Trop2 x CD3 / Dual CAB TCE / OncologyEpidermoid cancerPreclinical
- BA3362BA3362 / Nectin4 x CD3 / Dual CAB TCE / Oncology
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- Relacorilantglucocorticoid receptor / selective cortisol modulator / EndocrinologyHypercortisolism (GRACE)Phase IIIFiledHypercortisolism associated with adrenal adenoma or hyperplasia (GRADIENT)Phase IIIFiledLong-term safety extensionPhase IIFiled
- KorlymKorlym / glucocorticoid receptor / cortisol modulator / EndocrinologyPrevalence and treatment of hypercortisolism in difficult-to-control type 2 diabetes (CATALYST)Phase IV
- LifyorliLifyorli / glucocorticoid receptor / selective cortisol modulator / OncologyPlatinum-resistant ovarian cancer (ROSELLA)Phase IIIApproved
- Relacorilant + Nab-paclitaxel + Bevacizumabglucocorticoid receptor / selective cortisol modulator combination / OncologyPlatinum-resistant ovarian cancer (BELLA Arm A)Phase IIPlatinum-sensitive ovarian cancer (BELLA Arm B)Phase III
- Relacorilant + Nab-paclitaxelglucocorticoid receptor / selective cortisol modulator combination / OncologyEndometrial cancer (BELLA Arm C)Phase IICervical cancer (STELLA)Phase II
- Relacorilant + Nab-paclitaxel + Gemcitabineglucocorticoid receptor / selective cortisol modulator combination / OncologyPancreatic cancer (TRIDENT)Phase II
- Nenocorilant + Nivolumabglucocorticoid receptor / selective cortisol modulator combination / OncologySolid tumors (SYNERGY)Phase II
- Relacorilant + Enzalutamideglucocorticoid receptor / selective cortisol modulator combination / OncologyProstate cancerPhase II
- Dazucorilantglucocorticoid receptor / selective cortisol modulator / NeurologyALS (DAZALS)Phase II
- Miricorilantglucocorticoid receptor / selective cortisol modulator / MetabolismMASH (MONARCH)Phase II
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- FruquintinibHMPL-013 / ELUNATE / FRUZAQLA / VEGFR 1/2/3 / Small molecule / OncologyColorectal cancerApproved/MarketedApprovedChinaUSEuropeJapanGastric cancerPhase IIIActiveGlobalOther solid tumors (combination studies)ClinicalActiveGlobal
- SavolitinibHMPL-504 / AZD6094 / ORPATHYS / c-MET / Small molecule / OncologyNon-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 / OncologyNeuroendocrine tumorsApproved/MarketedApprovedChina
- SovleplenibHMPL-523 / Syk / Small molecule / Hematology/ImmunologyImmune thrombocytopenia (ITP)NDA/Regulatory reviewUnder regulatory reviewChina
- FanregratinibHMPL-453 / FGFR / Small molecule / OncologyCholangiocarcinoma (FGFR2 fusion)NDA/Regulatory reviewUnder regulatory reviewChina
- HMPL-760HMPL-760 / BTK (non-covalent) / Small molecule / Oncology/HematologyRelapsed/refractory diffuse large B-cell lymphoma (DLBCL)Phase IIIInitiatedChina
- HMPL-A251HMPL-A251 / HER2 (PI3K/PIKK payload) / Antibody-Targeted Therapy Conjugate (ATTC) / OncologyHER2-positive/HER2-low solid tumorsPhase IActiveChinaUS
- HMPL-A580HMPL-A580 / EGFR (PI3K/PIKK payload) / Antibody-Targeted Therapy Conjugate (ATTC) / OncologySolid tumorsPhase IInitiatedGlobal
- HMPL-A830HMPL-A830 / ATTC (undisclosed) / Antibody-Targeted Therapy Conjugate (ATTC) / OncologySolid tumorsPreclinical/Phase I plannedPlannedGlobal
- TazemetostatTAZVERIK / EZH2 / Small molecule / OncologyEpithelioid sarcoma / follicular lymphomaLicensed/CommercialLicensed product updateChina
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- tirzepatideMounjaro / Zepbound
- donanemabKisunla
- abemaciclibVerzenio
- pirtobrutinibJaypirca
- mirikizumabOmvoh
- lebrikizumabEbglyss
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- BerobenatidePF-06954522 / GLP-1/GIP / Small Molecule / Internal MedicineChronic Weight ManagementPhase 2bOngoingGlobal
- elranatamabPF-06863135 / ELREXFIO / BCMA x CD3 / Bispecific Antibody / OncologyDouble-class Exposed Relapsed / Refractory Multiple MyelomaPhase 3OngoingGlobal
- ritlecitinibPF-06651600 / LITFULO / JAK3/TEC / Small Molecule / Inflammation and ImmunologyVitiligoPhase 3OngoingGlobalModerate Alopecia AreataPhase 3OngoingGlobal
- marstacimabPF-06741086 / HYMPAVZI / TFPI / Monoclonal Antibody / Rare DiseaseHemophilia A/B with InhibitorsRegistrationOngoingGlobal
- MevrometostatPF-06821497 / EZH2 / Small Molecule / Oncology1-2L Metastatic Castration-resistant Prostate Cancer Post-abirateronePhase 3OngoingGlobal
- Sigvotatug vedotinPF-08046047 / Integrin beta-6 / Antibody-Drug Conjugate (ADC) / OncologyNon-Small Cell Lung Cancer (NSCLC)Phase 3OngoingGlobal
- VepdegestrantARV-471 / Estrogen Receptor (ER) / PROTAC Protein Degrader / OncologyER+/HER2- Metastatic Breast CancerPhase 3OngoingGlobal
- PCV25PF-07872412 / Pneumococcus / Vaccine / VaccinesPneumococcal InfectionPhase 3OngoingGlobal
- enfortumab vedotinPADCEV / Nectin-4 / Antibody-Drug Conjugate (ADC) / OncologyMuscle-invasive Bladder Cancer (Bladder Sparing)Phase 3OngoingGlobal
- Lyme Disease Vaccine CandidatePF-07307405 / Borrelia burgdorferi OspA / Vaccine / VaccinesLyme Disease InfectionPhase 3OngoingGlobal
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- ADG126ADG126 / muzastotug / CTLA-4 / fully human anti-CTLA-4 SAFEbody / Oncologyadvanced/metastatic solid tumorsPhase 1b/2dose expansion ongoing in combination with anti-PD-1 therapies
- ADG116ADG116 / CTLA-4 / fully human anti-CTLA-4 NEObody / Oncologyadvanced/metastatic solid tumorsPhase 1b/2ongoing
- ADG206ADG206 / CD137 / masked IgG1 Fc-engineered anti-CD137 POWERbody / Oncologyadvanced/metastatic tumorsPhase 1ongoing
- ADG106ADG106 / CD137 / fully human ligand-blocking agonistic anti-CD137 IgG4 mAb NEObody / Oncologyadvanced/metastatic solid tumors and/or relapsed/refractory non-Hodgkin's lymphomaPhase 1b/2ongoing
- ADG153ADG153 / CD47 / masked anti-CD47 IgG1 SAFEbody / Oncologyhematologic and solid tumorsIND-enablingpreclinical
- ADG138ADG138 / HER2 x CD3 / HER2xCD3 POWERbody T-cell engager / OncologyHER2-expressing solid tumorsIND-enablingpreclinical
- ADG152ADG152 / CD20 x CD3 / bispecific CD20xCD3 T-cell engager POWERbody / OncologyB-cell malignanciesIND-enablingpreclinical
- ADG104ADG104 / PD-L1 / antibody / OncologycancerPhase 2in development by Sanjin
- ADG125ADG125 / CSF-1R / antibody / OncologycancerPhase 1in development by Dragon Boat Biopharmaceutical
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- CASGEVYexa-cel / CASGEVY / BCL11A erythroid-specific enhancer / Non-viral ex vivo CRISPR/Cas9 gene-edited autologous cell therapy / HemoglobinopathiesSickle 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/metabolicSevere 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 / CardiovascularElevated 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 / CardiovascularElevated lipoprotein(a)IND/CTA-enablingActive
- CTX460CTX460 / SERPINA1 / In vivo SyNTase gene correction; LNP delivery / Rare diseaseAlpha-1 antitrypsin deficiencyPreclinical; clinical trial expected mid-2026Active
- CTX340CTX340 / AGT / In vivo CRISPR/Cas9 gene editing; LNP delivery / CardiovascularRefractory hypertensionIND/CTA-enabling; clinical trial expected H1 2026Active
- CTX611SRSD107 / Factor XI / FXI mRNA / Long-acting double-stranded siRNA / Thromboembolic/cardiovascular diseasesVenous 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; oncologySystemic 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 / diabetesType 1 diabetesPreclinical; progressing toward clinicActive
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- EVT8683BMS-986419 / eIF2b / NeuroscienceNeurodegeneration
- CNTX 6016CB2 / PainPain
- EVT801VEGFR3 / OncologyOncology
- EVT401P2X7 / ImmunologyInflammationAutoimmune
- EVT894Infectious DiseasesChikungunya
- EXS21546A2a / OncologyOncology
- DSP-11815-HT1A / NeuroscienceObsessive Compulsive Disorder (OCD)
- CT7001CDK7 / OncologyOncology
- TPM203ImmunologyPemphigus Vulgaris
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- DarovasertibIDE196 / PKC / Small Molecule / Oncology1L metastatic uveal melanoma (HLA-A*02:01 negative)Phase 2/3Positive topline; NDA initiationGlobalNeoadjuvant primary uveal melanomaPhase 2/3OngoingUS
- IDE397IDE397 / MAT2A / Small Molecule / OncologyMTAP-deleted solid tumorsPhase 1OngoingUS
- IDE849IDE849 / DLL3 / ADC / OncologyDLL3-expressing solid tumors (SCLC, NEC)Phase 1OngoingUS
- IDE161IDE161 / PARG / Small Molecule / OncologyHRD solid tumorsPhase 1RecruitingUS
- IDE034IDE034 / PTK7/B7H3 / Bispecific ADC / OncologyB7H3/PTK7 solid tumors (NSCLC, CRC, breast, ovarian)Phase 1OngoingUS
- IDE892IDE892 / PRMT5 / Small Molecule / OncologyMTAP-deleted solid tumorsPhase 1OngoingUS
- IDE574IDE574 / KAT6/7 / Small Molecule / OncologySolid tumors (breast, prostate, CRC, lung)Phase 1OngoingUS
- IDE275IDE275 / Werner Helicase (WRN) / Small Molecule / OncologyMSI-high solid tumorsPhase 1OngoingUS
- IDE705IDE705 / Pol Theta (POLQ) / Small Molecule / OncologyHRD solid tumorsPhase 1OngoingUS
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- BrensocatibBRINSUPRI / DPP1 / Small Molecule / Respiratory / ImmunologyNon-Cystic Fibrosis Bronchiectasis (NCFB)MarketedActiveUSEUHidradenitis SuppurativaPhase 2ActiveGlobal
- Amikacin Liposome Inhalation SuspensionARIKAYCE / Bacterial 30S ribosomal subunit / Liposomal Inhalation Suspension / Respiratory / Infectious DiseaseRefractory Mycobacterium avium complex (MAC) Lung DiseaseMarketedActiveUSEUJPMAC Lung Disease (Label Expansion)Phase 3ActiveGlobal
- Treprostinil Palmitil Inhalation PowderTPIP / Prostacyclin receptor / Inhalation Powder / Respiratory / PulmonaryPulmonary 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 / ImmunologyInterstitial Lung Disease (ILD)Phase 2ActiveGlobalModerate-to-severe asthmaPhase 2ActiveGlobal
- INS1033INS1033 / Unknown / Unknown / Immunology & InflammationInflammatory diseasesPreclinicalActiveGlobal
- Intrathecal gene therapy for DMDDystrophin / Gene Therapy / Neurology / Rare DiseaseDuchenne Muscular Dystrophy (DMD)Phase 1ActiveGlobal
- INS1202INS1202 / Unknown / Unknown / Neurology / Rare DiseaseAmyotrophic Lateral Sclerosis (ALS)Phase 1ActiveGlobal
- Stargardt Disease Gene TherapyUnknown / Gene Therapy / Ophthalmology / Rare DiseaseStargardt DiseasePreclinicalActiveGlobal
- Next-Gen DPP1 InhibitorsDPP1 / Small Molecule / Immunology & InflammationUndisclosedPreclinicalActiveGlobal
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- ZanidatamabHER2 / Bispecific Antibody / OncologyBiliary Tract Cancers (BTC)Gastroesophageal Adenocarcinoma (GEA)Colorectal Cancer (CRC)
- LurbinectedinZepzelca / DNA alkylating / Small Molecule / OncologySmall Cell Lung Cancer (SCLC)Ewing Sarcoma
- DordaviproneModeyso / ClpP agonist / DRD2 antagonist / Small Molecule / OncologyH3 K27M-mutant diffuse glioma
- Daunorubicin and cytarabineJZP351 / Vyxeos / Vyxeos Liposomal / Topoisomerase inhibitor / nucleoside metabolic inhibitor / Liposomal Combination / OncologyMyelodysplastic Syndromes (MDS)Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)
- JZP898JZP898 / IFNα2b cytokine pro-drug / Biologic / OncologySolid Tumors
- JZP3507JZP3507 / ClpP agonist / DRD2 antagonist / Small Molecule / OncologyOncology
- JZP815JZP815 / pan-RAF / Small Molecule / OncologySolid TumorsHematologic Malignancies
- CannabidiolEpidyolex / Cannabinoid Receptors / Small Molecule / NeuroscienceLennox-Gastaut Syndrome (LGS)Dravet Syndrome (DS)Tuberous Sclerosis Complex (TSC)
- JZP441JZP441 / Orexin-2 receptor agonist / Small Molecule / NeuroscienceNarcolepsy Type 1Idiopathic Hypersomnia
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- pembrolizumabMK-3475 / Keytruda
- belzutifanMK-6482 / Welireg
- sotaterceptMK-7962
- sacituzumab tirumotecanMK-2870
- efinopegdutideMK-6024
- gocatamigMK-6070
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- Isaralgagene civaparvovecST-920 / alpha-galactosidase A (GLA) / Gene Therapy (AAV) / Metabolic DiseasesFabry DiseaseBLA/FilingActiveUS
- Giroctogene fitelparvovecSB-525 / Factor VIII / Gene Therapy (AAV) / HematologyHemophilia APhase 3ActiveUS
- ST-503ST-503 / Nav1.7 (SCN9A) / Gene Therapy (Zinc Finger Repressor, AAV9) / NeurologySmall Fiber Neuropathy (Chronic Neuropathic Pain)Phase 1/2ActiveUS
- ST-506ST-506 / Prion protein (PRNP) / Gene Therapy (Zinc Finger Repressor, STAC-BBB AAV) / NeurologyPrion DiseasePreclinicalActiveUS
- TX200TX200 / HLA-A2 / Cell Therapy (Autologous CAR-Treg) / TransplantationPrevention of Immune-Mediated Rejection in HLA-A2 Mismatched Kidney TransplantationPhase 1/2ActiveUSEurope
- IL23R CAR-TregIL23R / Cell Therapy (CAR-Treg) / AutoimmunityInflammatory Bowel Disease (Crohn's Disease)PreclinicalActiveGlobal
- Genentech Tau ProgramTau / Gene Therapy (Zinc Finger Epigenetic Regulator) / NeurologyTauopathies / Alzheimer's DiseasePreclinicalActiveGlobal
- Takeda Huntington's ProgramHTT / Gene Therapy (Zinc Finger Epigenetic Regulator) / NeurologyHuntington's DiseasePreclinicalActiveGlobal
- Alexion C9ORF72 ProgramC9ORF72 / Gene Therapy (Zinc Finger Transcription Factor) / NeurologyAmyotrophic Lateral Sclerosis (ALS)PreclinicalActiveGlobalFrontotemporal Lobar Degeneration (FTLD)PreclinicalActiveGlobal
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- Olanzapine LAITEV-'749
- DuvakitugAnti-TL1A
- DARI (ICS/SABA)TEV-'248
- EmrusolminTEV-'286
- Anti-IL-15TEV-'408
- Anti-PD1-IL2TEV-'278
- Sery433TEV-'2871
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- SuzetrigineVX-548 / Journavx / NaV1.8 / Small Molecule / PainModerate-to-Severe Acute PainPainful Diabetic Peripheral Neuropathy
- VX-993VX-993 / NaV1.8 / Small Molecule / PainPainful Diabetic Peripheral NeuropathyModerate-to-Severe Acute Pain
- Vanzacaftor/tezacaftor/deutivacaftorCFTR / Small Molecule / Cystic FibrosisCystic Fibrosis
- VX-522VX-522 / CFTR / mRNA / Cystic FibrosisCystic Fibrosis (non-responsive to modulators)
- InaxaplinVX-147 / APOL1 / Small Molecule / Kidney DiseaseAPOL1-mediated Kidney Disease (AMKD)
- VX-407VX-407 / PC1 / Small Molecule / Kidney DiseaseAutosomal Dominant Polycystic Kidney Disease (ADPKD)
- VX-880VX-880 / Islet Cell / Cell Therapy / Type 1 DiabetesType 1 Diabetes
- VX-264VX-264 / Islet Cell / Cell Therapy / Type 1 DiabetesType 1 Diabetes
- PovetaciceptALPN-303 / BAFF/APRIL / Biologic / Autoimmune / Kidney DiseaseIgA Nephropathy
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- XmAb819XmAb819 / ENPP3 x CD3 / Bispecific Antibody / OncologyClear Cell Renal Cell Carcinoma (ccRCC)Phase 1ActiveGlobalColorectal Cancer (CRC)Phase 1ActiveGlobalNon-Small Cell Lung Cancer (NSCLC)Phase 1ActiveGlobal
- XmAb541XmAb541 / CLDN6 x CD3 / Bispecific Antibody / OncologyOvarian CancerPhase 1ActiveGlobalGerm Cell Tumors (GCT)Phase 1ActiveGlobalSolid TumorsPhase 1ActiveGlobal
- XmAb808XmAb808 / B7-H3 x CD28 / Bispecific Antibody / OncologyProstate Cancer / Solid TumorsPhase 1ActiveGlobal
- XmAb942XmAb942 / TL1A / Monoclonal Antibody / ImmunologyUlcerative Colitis (UC)Phase 2ActiveGlobalInflammatory Bowel Disease (IBD)Phase 2ActiveGlobal
- XmAb412XmAb412 / TL1A x IL23p19 / Bispecific Antibody / ImmunologyInflammatory Bowel Disease (IBD)Phase 1ActiveGlobal
- PlamotamabCD20 x CD3 / Bispecific Antibody / ImmunologyRheumatoid ArthritisPhase 1ActiveGlobal
- XmAb657XmAb657 / CD19 x CD3 / Bispecific Antibody / ImmunologyIdiopathic Inflammatory MyositisPhase 1ActiveGlobal
- ObexelimabCD19 x FcγRIIb / Monoclonal Antibody / ImmunologyAutoimmune DiseasesPhase 3ActiveGlobal
- TobevibartHepatitis B Virus / Monoclonal Antibody / Infectious DiseaseChronic 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/ImmunologyAnaphylaxis/allergic reactionsdevelopment
- AMP-105AMP-105 / peptide / OncologyPoorly treated cancersdevelopmentUnited StatesCanada
- AMP-107AMP-107 / VEGFR / topical eye-drop peptide / OphthalmologyWet age-related macular degenerationdevelopmentUnited StatesCanada
- AMP-109AMP-109 / peptide-docetaxel conjugate / OncologyCancerdevelopmentUnited StatesCanada
- AMP-110AMP-110 / ACTH receptor pathway / synthetic human corticotropin analog peptideMultiple indicationsdevelopmentUnited StatesCanada
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- PaltusotineCRN00808 / PALSONIFY / SST2 / oral nonpeptide SST2 agonist / Endocrinology; oncologyAcromegaly (US)ApprovedapprovedUSAcromegaly (EU)Registrationalunder reviewEUCarcinoid syndrome associated with NETsPhase 3clinicalGlobal
- AtumelnantCRN04894 / MC2R/ACTH receptor / oral nonpeptide ACTH receptor antagonist / EndocrinologyCongenital adrenal hyperplasia (adult)Phase 3clinicalCongenital adrenal hyperplasia (pediatric)Phase 2/3clinicalACTH-dependent Cushing's syndromePhase 2/3planned
- CRN09682CRN09682 / SST2 / nonpeptide drug conjugate; MMAE payload / OncologySST2-expressing NETs and other SST2-expressing solid tumorsPhase 1/2clinical
- PTH AntagonistPTH receptor / oral nonpeptide antagonist / EndocrinologyHyperparathyroidism and other diseases of hypercalcemiaPreclinicalIND-enabling
- TSH AntagonistTSH receptor / oral nonpeptide antagonist / EndocrinologyGraves' disease; thyroid eye diseasePreclinicalIND-enabling
- SST3 AgonistSST3 / oral nonpeptide agonist / NephrologyPolycystic kidney diseasePreclinicalIND-enabling
- Oral GLP-1 NonpeptideGLP-1 receptor / oral nonpeptide agonist / Metabolic diseaseDiabetes; obesityPreclinicaldisclosed
- Oral GIP NonpeptideGIP receptor / oral nonpeptide agonist / Metabolic diseaseDiabetes; obesityPreclinicaldisclosed
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- CVGBMCVGBM / tumor-associated antigens / unmodified mRNA cancer precision immunotherapy / Oncologynewly 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 / Oncologysquamous 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 diseaseCOVID-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 diseaseCOVID-19Phase 2study completed August 2024; GSK assumed full control
- seasonal influenza mRNA vaccine candidateinfluenza antigens / multivalent mRNA vaccine / Infectious diseaseseasonal influenzaPhase 2GSK assumed full control
- avian influenza H5N1 mRNA vaccine candidateH5N1 influenza antigens / mRNA vaccine / Infectious diseaseavian influenza H5N1Phase 2GSK assumed full control
- COVID-19/influenza combination mRNA vaccine candidateSARS-CoV-2 and influenza antigens / combination mRNA vaccine / Infectious diseaseCOVID-19 and influenzaPhase 1/2GSK initiated study November 2024; GSK assumed full control
- UPEC FimH ferritin nanoparticle mRNA vaccine candidateFimH / mRNA vaccine / Infectious diseaseurinary tract infections caused by uropathogenic E. coliPreclinicallead candidate identified
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- EVX-01EVX-01 / Personalized neoantigens / Liposomal/peptide personalized cancer vaccine / CancerAdvanced melanomaPhase 2
- EVX-03EVX-03 / Personalized neoantigens and endogenous retrovirus antigens / Targeted DNA personalized cancer vaccine / CancerSolid tumorsPreclinicalCTA/IND ready
- EVX-04EVX-04 / Tumor-specific antigens derived from endogenous retrovirus elements / DNA off-the-shelf therapeutic cancer vaccine / CancerAcute myeloid leukemiaPreclinicalPlanned first-in-human study
- EVX-B1EVX-B1 / S. aureus toxins and virulence factors / Protein multi-component prophylactic vaccine / Infectious diseasesS. aureus skin and soft tissue infectionsPreclinical
- EVX-B2EVX-B2 / N. gonorrhoeae antigens / Protein multi-component prophylactic vaccine / Infectious diseasesGonorrheaPreclinical
- EVX-B2 mRNAEVX-B2 / N. gonorrhoeae antigens / mRNA multi-component prophylactic vaccine / Infectious diseasesGonorrheaPreclinical
- EVX-B4EVX-B4 / Group A Streptococcus antigens / Prophylactic vaccine / Infectious diseasesGroup A Streptococcus infectionTarget discovery
- EVX-V1EVX-V1 / CMV B- and T-cell antigens; pre-fusion glycoprotein B construct / Multi-component prophylactic vaccine / Infectious diseasesCytomegalovirus infectionPreclinical
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- ZanzalintinibXL092 / MET/VEGFR/AXL/MER / Next-generation TKI / OncologyColorectal 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 / OncologyMultiple solid tumorsPhase 1XL309 or XL309 + olaparib
- XB010XB010 / 5T4 / Next-generation 5T4-MMAE ADC / OncologyMultiple solid tumorsPhase 1XB010 or XB010 + pembrolizumab
- XB628XB628 / PD-L1 + NKG2A / Bispecific antibody / OncologyMultiple solid tumorsPhase 1Ongoing clinical study
- XB371XB371 / Tissue factor / Next-generation TF-TOPOi ADC / OncologyMultiple solid tumorsPhase 1Ongoing clinical study
- ADU-1805ADU-1805 / SIRPα / SIRPα-targeting mAb / OncologyMultiple solid tumorsPhase 1ADU-1805 or ADU-1805 + pembrolizumab
- XB773XB773 / DLL3 / DLL3-TOPOi ADC / OncologyNot disclosedDiscovery/PreclinicalDiscovery/Preclinical
- SSTR2 AgonistSomatostatin receptor subtype 2 / SSTR2 agonist program development candidate / OncologyNot disclosedDiscovery/PreclinicalDiscovery/Preclinical
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- TebentafuspKIMMTRAK / gp100 (HLA-A*02) / ImmTAC (TCR bispecific) / Oncologymetastatic uveal melanomaApprovedmarketedUSEUadjuvant uveal melanomaPhase 3ongoing (ATOM, EORTC-sponsored)2L+ advanced cutaneous melanomaPhase 3ongoing (TEBE-AM)
- BrenetafuspIMC-F106C / PRAME (HLA-A*02) / ImmTAC (TCR bispecific) / Oncology1L 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) / Oncologymultiple solid tumorsPhase 1ongoing
- IMC-R117CIMC-R117C / PIWIL1 (HLA-A*02) / ImmTAC (TCR bispecific) / Oncologycolorectal and GI cancersPhase 1/2ongoing
- IMC-M113VIMC-M113V / Gag (HLA-A*02) / ImmTAV (TCR bispecific) / Infectious DiseaseHIVPhase 1ongoing
- IMC-I109VIMC-I109V / Envelope (HLA-A*02) / ImmTAV (TCR bispecific) / Infectious DiseaseHBVclinical development
- IMC-S118AIIMC-S118AI / PPI x PD1 (HLA-A*02) / ImmTAAI (TCR bispecific) / Autoimmune Diseasetype 1 diabetesPhase 1ongoing
- IMC-U120AIIMC-U120AI / CD1a x PD1 (non-HLA restricted) / ImmTAAI (TCR bispecific) / Autoimmune Diseaseatopic dermatitisIND-enabling
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- LacutamabKIR3DL2 / Monoclonal antibody / OncologySézary syndromeMycosis fungoidesCutaneous T cell lymphomas
- IPH4502IPH4502 / Nectin-4 / Antibody-drug conjugate (ADC) / OncologySolid tumors
- MonalizumabNKG2A / Monoclonal antibody / OncologyNon-small cell lung cancer (NSCLC)
- IPH6501IPH6501 / CD20 / NK cell engager (ANKET) / OncologyB-cell Non-Hodgkin's Lymphoma
- IPH6101IPH6101 / CD123 / NK cell engager (ANKET) / Oncology / AutoimmuneHematologic malignanciesAutoimmune indications
- IPH6401IPH6401 / BCMA / NK cell engager (ANKET) / OncologyMultiple myeloma
- IPH5201IPH5201 / CD39 / Monoclonal antibody / OncologySolid tumors
- IPH5301IPH5301 / CD73 / Monoclonal antibody / OncologySolid tumors
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- OPGx-LCA5OPGx-LCA5 / LCA5 / AAV Gene Therapy / OphthalmologyLeber Congenital Amaurosis Type 5 (LCA5)
- OPGx-BEST1OPGx-BEST1 / BEST1 / AAV Gene Therapy / OphthalmologyBest Vitelliform Macular Dystrophy (BVMD)Autosomal Recessive Bestrophinopathy (ARB)
- OPGx-RDH12OPGx-RDH12 / RDH12 / AAV Gene Therapy / OphthalmologyRDH12-associated Leber Congenital Amaurosis (RDH12-LCA)
- OPGx-MERTKOPGx-MERTK / MERTK / AAV Gene Therapy / OphthalmologyMERTK-associated Retinitis Pigmentosa
- OPGx-RHOOPGx-RHO / RHO / AAV Gene Therapy / OphthalmologyAutosomal Dominant Retinitis Pigmentosa (RHO-adRP)
- OPGx-CNGB1OPGx-CNGB1 / CNGB1 / AAV Gene Therapy / OphthalmologyCNGB1-associated Inherited Retinal Diseases
- OPGx-NMNAT1OPGx-NMNAT1 / NMNAT1 / AAV Gene Therapy / OphthalmologyNMNAT1-associated Inherited Retinal Diseases
- Phentolamine Ophthalmic Solution 0.75%Small Molecule / OphthalmologyPharmacologically induced mydriasisPresbyopiaLow-light visual disturbances following keratorefractive surgery
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- KT-621KT-621 / STAT6 / Targeted Protein Degrader / Immunology / RespiratoryAtopic DermatitisPhase 2bOngoingGlobalAsthmaPhase 2bOngoingGlobal
- KT-579KT-579 / IRF5 / Targeted Protein Degrader / ImmunologyAutoimmune Diseases (Lupus, Rheumatoid Arthritis, IBD)Phase 1PlannedGlobal
- KT-295KT-295 / TYK2 / Targeted Protein Degrader / ImmunologyInflammatory Bowel Disease (IBD), PsoriasisPhase 1OngoingGlobal
- KT-485SAR447971 / IRAK4 / Targeted Protein Degrader / ImmunologyImmuno-inflammatory diseasesPreclinicalOngoingGlobal
- KT-200KT-200 / CDK2 / Molecular Glue Degrader / OncologyBreast Cancer and other Solid TumorsPreclinicalOngoingGlobal
- KT-333KT-333 / STAT3 / Targeted Protein Degrader / OncologyHematological Malignancies and Solid TumorsPhase 1OngoingGlobal
- KT-253KT-253 / MDM2 / Targeted Protein Degrader / OncologyHigh-Grade Myeloid Malignancies and Solid TumorsPhase 1OngoingGlobal
- KT-474SAR444656 / IRAK4 / Targeted Protein Degrader / ImmunologyHidradenitis Suppurativa (HS), Atopic DermatitisPhase 2bDiscontinuedGlobal
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- sepiapterinSephience
- atalurenTranslarna
- deflazacortEmflaza
- eladocagene exuparvovecUpstaza / Kebilidi
- votoplamPTC518
- utreloxastatPTC857
- risdiplamEvrysdi
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- fenebrutinibBTK / Small molecule / NeuroscienceMultiple Sclerosis
- petrelintideAmylin / Peptide / Cardiovascular, Renal & MetabolismObesity
- CT-996CT-996 / GLP-1 / Small molecule / Cardiovascular, Renal & MetabolismObesity
- giredestrantEstrogen receptor (SERD) / Small molecule / OncologyEarly and metastatic breast cancer
- trontinemabAmyloid beta / TfR / Bispecific antibody / NeuroscienceAlzheimer's Disease
- NXT007NXT007 / Factor IX/X / Bispecific antibody / HematologyHemophilia A
- afimkibartBiologic / ImmunologyImmunological disorders
- RVT-3101RVT-3101 / TL1A / Monoclonal antibody / ImmunologyInflammatory Bowel Disease
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- ecopipamNeurologyTourette's syndrome
- TEV-'749TEV-'749 / NeurologySchizophrenia
- deucrictibantRare DiseaseHereditary angioedema
- daraxonrasibOncologyPancreatic cancer
- litifilimabImmunologyLupus
- pelacarsenCardiovascularCardiovascular disease
- zidesamtinibROS1 / OncologyROS1 mutation-positive non-small cell lung cancer
- JNJ-4804JNJ-4804 / ImmunologyAutoimmune diseases
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- SION-719SION-719 / NBD1 (Nucleotide Binding Domain 1 of CFTR) / Small Molecule / Respiratory / Genetic DiseasesCystic FibrosisPhase 2aActive
- SION-451SION-451 / NBD1 (Nucleotide Binding Domain 1 of CFTR) / Small Molecule / Respiratory / Genetic DiseasesCystic FibrosisPhase 1Active
- SION-638SION-638 / NBD1 (Nucleotide Binding Domain 1 of CFTR) / Small Molecule / Respiratory / Genetic DiseasesCystic FibrosisPhase 1Active
- SION-109SION-109 / ICL4 (Intracellular Loop 4 of CFTR) / Small Molecule / Respiratory / Genetic DiseasesCystic FibrosisPhase 1Active
- GalicaftorABBV-2222 / TMD1-directed CFTR corrector / Small Molecule / Respiratory / Genetic DiseasesCystic FibrosisPhase 2Active
- NavocaftorABBV-3067 / CFTR potentiator / Small Molecule / Respiratory / Genetic DiseasesCystic FibrosisPhase 2Active
- ABBV-2851ABBV-2851 / TMD1-directed CFTR corrector / Small Molecule / Respiratory / Genetic DiseasesCystic FibrosisPhase 1Active
- SION-676SION-676 / TMD1-directed CFTR corrector / Small Molecule / Respiratory / Genetic DiseasesCystic FibrosisPreclinicalActive
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- SRP-1001SRP-1001 / FSHD1 / siRNA / NeuromuscularFacioscapulohumeral muscular dystrophy, Type 1 (FSHD1)
- SRP-1003SRP-1003 / DM1 / siRNA / NeuromuscularMyotonic dystrophy, Type 1 (DM1)
- SRP-1002SRP-1002 / siRNA / PulmonaryIdiopathic pulmonary fibrosis (IPF)
- SRP-1004SRP-1004 / siRNA / NeurologySpinocerebellar ataxia type 2 (SCA2)
- SRP-1005SRP-1005 / siRNA / NeurologyHuntington's disease (HD)
- SRP-1007SRP-1007 / siRNA / NeurologySpinocerebellar ataxia type 1 (SCA1)
- SRP-1006SRP-1006 / siRNA / NeurologySpinocerebellar ataxia type 3 (SCA3)
- bidridistrogene xeboparvovecSRP-9003 / LGMD2E/R4 β-sarcoglycan / Gene Therapy / NeuromuscularLimb-girdle Muscular Dystrophy
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- ZanidatamabZW25 / Ziihera / HER2 / Bispecific Antibody / OncologyBiliary Tract Cancer (BTC)ApprovedActiveUSEuropeGastroesophageal Adenocarcinoma (GEA)Phase 3ActiveGlobalBreast Cancer (Neoadjuvant and Adjuvant)Phase 2ActiveGlobal
- ZW191ZW191 / FRα / Antibody-Drug Conjugate (ADC) / OncologyAdvanced Solid Tumors (Ovarian/Gynecological and Lung/Thoracic)Phase 1ActiveGlobal
- ZW251ZW251 / GPC3 / Antibody-Drug Conjugate (ADC) / OncologyHepatocellular Carcinoma (HCC) / Solid TumorsPhase 1ActiveGlobal
- ZW220ZW220 / NaPi2b / Antibody-Drug Conjugate (ADC) / OncologyGynecological and Thoracic CancersPreclinicalActiveGlobal
- ZW209ZW209 / DLL3 x CD3 x CD28 / Trispecific T-Cell Engager / OncologyThoracic CancersPreclinicalActiveGlobal
- ZW1528ZW1528 / IL-4Rα x IL-33 / Bispecific Antibody / Autoimmune / Inflammatory DiseasesRespiratory InflammationPreclinicalActiveGlobal
- PasritamigJNJ-78278343 / CD3 / Prostate Target / Bispecific Antibody / OncologySolid TumorsClinicalActiveGlobal
- DS-2243DS-2243 / T-Cell Engager / Bispecific T-Cell Engager / OncologyAdvanced Solid TumorsPhase 1ActiveGlobal
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- OlverembatinibHQP1351 / BCR-ABL / small molecule TKI / OncologyCML-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 / OncologyCLL/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 / OncologyALK-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 / OncologyUnresectable/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 / OncologyEGFR-mutant NSCLCPhase 1bOngoing; +osimertinibChinaR/R NHLPhase 1b/2OngoingChinaRecurrent ovarian and endometrial cancersPhase 1Ongoing; +cobimetinib
- APG-5918APG-5918 / EED/PRC2 / small molecule inhibitor / Oncology; HematologyAdvanced solid tumors and hematologic malignanciesPhase 1OngoingChinaUnited StatesAnemia-related indicationsPhase 1OngoingChina
- APG-3288APG-3288 / BTK / PROTAC degrader / OncologyR/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 / NeuropsychiatryTreatment-resistant depressionPhase 2bactive
- ALTO-300MT1/2 & 5HT2C / oral small molecule / NeuropsychiatryMajor depressive disorderPhase 2bactiveUnited States
- ALTO-100BDNF/neural plasticity / oral small molecule / NeuropsychiatryBipolar depressionPhase 2bactive
- ALTO-101PDE4 / transdermal PDE4 inhibitor / NeuropsychiatryCognitive impairment associated with schizophreniaPhase 2active
- ALTO-203Histamine H3 receptor / oral small molecule inverse agonist / NeuropsychiatryMajor depressive disorder with anhedoniaPhase 2aactive
- ALTO-202NMDA NR2B/GluN2B / oral small molecule negative allosteric modulator / NeuropsychiatryMajor depressive disorderactive
- ALTO-208CTC 413 / D3/D2 & NK-1 / fixed-dose combination of pramipexole and aprepitant / NeurologyParkinson's diseaseactive
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- ARV-102ARV-102 / LRRK2 / oral brain-penetrant PROTAC degrader / NeurologyParkinson’s diseasePhase 1/1bunder investigationProgressive supranuclear palsyunder investigation
- ARV-027ARV-027 / polyQ-AR / oral peripherally restricted PROTAC degrader / NeurologySpinal-bulbar muscular atrophyPhase 1/1bunder investigation
- ARV-806ARV-806 / KRAS G12D / PROTAC degrader / OncologyPancreatic cancerPhase 1/1bunder investigationColorectal cancerPhase 1/1bunder investigationNon-small cell lung cancerPhase 1/1bunder investigation
- ARV-393ARV-393 / BCL6 / oral PROTAC degrader / OncologyNon-Hodgkin lymphoma / B-cell malignanciesPhase 1/1bunder investigation
- ARV-6723ARV-6723 / HPK1 / oral PROTAC degrader / OncologyAdvanced solid tumorsPreclinicalunder investigation
- VepdegestrantARV-471 / ER / oral PROTAC estrogen receptor degrader / OncologyER+/HER2- locally advanced or metastatic breast cancerPhase 3under investigation
- LuxdegalutamideARV-766; JSB462 / AR / oral PROTAC androgen receptor degrader / OncologyProstate cancerPhase 2under investigation
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- epcoritamabGEN3013 / EPKINLY/TEPKINLY / CD20xCD3 / Bispecific antibody / OncologyRelapsed 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) / OncologyPlatinum-resistant ovarian cancerPhase 3ActiveGlobalAdvanced endometrial cancerPhase 1/2ActiveGlobal
- petosemtamabMCLA-158 / EGFRxLGR5 / Bispecific antibody / OncologyFirst-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 / OncologyMultiple myeloma (multiple lines)ApprovedMarketedGlobal
- ofatumumabKesimpta/Arzerra / CD20 / Monoclonal antibody / Neurology/OncologyRelapsing multiple sclerosis (Kesimpta)ApprovedMarketedGlobalChronic lymphocytic leukemia (Arzerra)ApprovedMarketedUSEU
- tisotumab vedotinTivdak / Tissue factor / Antibody-drug conjugate (ADC) / OncologyRecurrent or metastatic cervical cancerApprovedMarketedUSEUJapan
- HexaBody-CD38GEN3014 / CD38 / HexaBody monoclonal antibody / OncologyMultiple myelomaPhase 1/2ActiveGlobal
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- AleniglipronGSBR-1290 / GLP-1R / Oral small molecule agonist / Metabolic / ObesityObesity / 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 / ObesityObesityPhase 1Active (SAD study initiated Dec 2025)Global
- ACCG-3535ACCG-3535 / Amylin/calcitonin receptor (DACRA) / Oral small molecule agonist / Metabolic / ObesityObesityPreclinical / IND-enablingPhase 1 initiation expected 2H 2026Global
- ANPA-0073ANPA-0073 / Apelin (APJ) receptor / Oral small molecule biased agonist / Metabolic / ObesityObesity (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 / FibrosisIdiopathic pulmonary fibrosis (IPF)Phase 1Phase 1 SAD/MAD completedGlobal
- GIPR ProgramGIPR (and GLP-1R/GIPR) / Oral small molecule (agonist/antagonist) / Metabolic / ObesityObesity and related diseasesDiscovery / PreclinicalActiveGlobal
- GCGR ProgramGCGR (and GLP-1R/GCGR) / Oral small molecule agonist / Metabolic / ObesityObesity and related diseasesDiscovery / PreclinicalActiveGlobal
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- PitolisantWAKIX / histamine-3 receptor (H3R) antagonist/inverse agonist / small molecule / Rare neurological disordersPrader-Willi syndromePhase 3USNarcolepsy (GR formulation)Phase 3USNarcolepsy (HD formulation)Phase 3US
- BP1.15205orexin-2 receptor (OX2R) agonist / small molecule / Sleep/wake disordersNarcolepsy and other sleep/wake disordersPhase 1USLatin America
- EPX-1005-HT (serotonin) receptor agonist / small molecule / Rare epilepsiesDravet syndromePhase 3USLennox-Gastaut syndromePhase 3US
- EPX-200selective 5HT2C agonist / small molecule / Rare epilepsiesDevelopmental and epileptic encephalopathiesPreclinicalIND-enablingUS
- CBS-105cell therapy / Rare neurological disordersTreatment-resistant narcolepsyPreclinicalUS
- HBS-102MCHR1 antagonist / small molecule / Rare neurological disordersPrader-Willi syndromePreclinicalUS
- CBS-104cell therapy / Rare epilepsiesRefractory epilepsyPreclinicalUS
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- LifileucelLN-144 / Amtagvi / Polyclonal Neoantigens / Tumor Infiltrating Lymphocyte (TIL) Therapy / OncologyMelanomaNon-Small Cell Lung Cancer (NSCLC)
- TIL CombinationsPolyclonal Neoantigens / Cell Therapy Combination / OncologySolid Tumors
- PD-1 Selected TILPolyclonal Neoantigens / PD-1+ / Tumor Infiltrating Lymphocyte (TIL) Therapy / OncologySolid Tumors
- Third Generation (Gen 3) TILPolyclonal Neoantigens / Tumor Infiltrating Lymphocyte (TIL) Therapy / OncologySolid Tumors
- PBL TherapyPolyclonal Neoantigens / Peripheral Blood Lymphocyte Therapy / OncologySolid Tumors
- PD-1 Inactivated TILIOV-4001 / PD-1 / Genetically Modified Cell Therapy / OncologySolid Tumors
- Modified IL-2 Fusion ProteinIOV-3001 / IL-2 Receptor / Antibody Cytokine Engrafted Protein / OncologyMelanomaSolid Tumors
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- LX2006LX2006 / FXN / AAV gene therapy / CardiovascularFriedreich's ataxia (FA) cardiomyopathy
- LX2020LX2020 / PKP2 / AAV gene therapy / CardiovascularPKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM)
- LX2021LX2021 / Cx43 / AAV gene therapy / CardiovascularDSP (Desmoplakin) cardiomyopathyDilated cardiomyopathy
- LX2022LX2022 / TNNI3 / AAV gene therapy / CardiovascularHypertrophic cardiomyopathy (HCM)
- LX1001LX1001 / APOE2 / AAV gene therapy / NeurologyAPOE4-associated Alzheimer's disease
- LX1021LX1021 / Christchurch-modified APOE2 / AAV gene therapy / NeurologyAPOE4-associated Alzheimer's disease
- LX1020LX1020 / APOE2 / APOE4 (miRNA) / AAV gene therapy / NeurologyAPOE4-associated Alzheimer's disease
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- insulin humanAfrezza / Insulin receptor / Recombinant Protein / EndocrineType 1 and Type 2 Diabetes MellitusMarketedApprovedUSBrazilPediatric Diabetes MellitusPhase 3OngoingUS
- treprostinilTyvaso DPI / Prostacyclin receptor / Small Molecule / Orphan Lung DiseasesPulmonary 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 DiseasesNontuberculous Mycobacterial (NTM) Lung DiseasePhase 3DiscontinuedUS
- clofazimine DPIMNKD-102 / Bacterial DNA / Mycobacterial growth / Small Molecule / Orphan Lung DiseasesNontuberculous Mycobacterial (NTM) Lung DiseasePreclinicalActiveUS
- nintedanib DPIMNKD-201 / Tyrosine kinase / Small Molecule / Orphan Lung DiseasesIdiopathic Pulmonary Fibrosis (IPF)Phase 1CompletedUS
- dornase alfa DPIMNKD-301 / DNase I / Recombinant Protein / Orphan Lung DiseasesCystic FibrosisPreclinicalActiveUS
- FBM5712 DPIMNKD-501 / ALK-5 kinase / TGF-beta / Small Molecule / Orphan Lung DiseasesPulmonary FibrosisPreclinicalActiveUS
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- SomatrogonhGH-CTP / NGENLA / Growth hormone receptor / Biologic / EndocrinologyGrowth Hormone Deficiency (Pediatric)ApprovedactiveUSEUJapanGlobalGrowth Hormone Deficiency (Adults)Phase 3activeGlobal
- Calcifediol extended-releaseRayaldee / Rayaldee / Vitamin D receptor / Small Molecule / NephrologySecondary Hyperparathyroidism in Stage 5 Chronic Kidney Disease (Hemodialysis)Phase 2activeUS
- MDX2004MDX2004 / CD3 x CD28 x 4-1BB / Multispecific Antibody / OncologyAdvanced CancersPhase 1/2aactiveUS
- MDX2003MDX2003 / T-cell engager / Multispecific Antibody / OncologyLymphomaPhase 1activeUS
- ModeX COVID-19 Multispecific AntibodySARS-CoV-2 / Multispecific Antibody / Infectious DiseaseCOVID-19PreclinicalactiveUS
- ModeX Influenza Multispecific AntibodyInfluenza virus / Multispecific Antibody / Infectious DiseaseInfluenzaPreclinicalactiveUS
- Oral GLP-2 AgonistGLP-2 / Peptide / GastroenterologyShort Bowel SyndromePreclinicalactiveGlobal
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- 18F-PivalateRAD 101 / FASN (Fatty acid synthase) / Small Molecule Radiopharmaceutical / DiagnosticsBrain metastases from solid tumors (Imaging)Phase 2bEnrollingUS
- 177Lu-RAD202RAD 202 / HER2 / Nanobody Radiotherapeutic / OncologyHER2-positive advanced solid tumorsPhase 1ActiveUSGlobal
- 177Lu-RAD204RAD 204 / PD-L1 / Nanobody Radiotherapeutic / OncologyPD-L1 positive advanced solid tumorsPhase 1ActiveUSGlobal
- 68Ga-TrivehexinRAD 301 / αvβ6-integrin / Peptide Radiopharmaceutical / DiagnosticsLoco-regional pancreatic cancer (Imaging)Phase 2ActiveGlobal
- 177Lu-D0301RAD 302 / αvβ6-integrin / Peptide Radiotherapeutic / OncologySolid tumors (PDAC, NSCLC, HNSCC)PreclinicalActiveGlobal
- BetaBartRV-01 / B7-H3 / Radiotherapeutic / OncologyB7H3-expressing solid tumorsPhase 1/2aActiveUS
- 161Tb-RAD 402RAD 402 / KLK3 (PSA) / Monoclonal Antibody Radiotherapeutic / OncologyAdvanced prostate cancerPhase 1ActiveAustralia
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- SGT-003SGT-003 / Gene Therapy / NeuromuscularDuchenne muscular dystrophy (INSPIRE)Phase 1/2ActiveGlobalDuchenne muscular dystrophy (IMPACT)Phase 3Activeex-U.S.
- SGT-212SGT-212 / Gene Therapy / NeuromuscularFriedreich's ataxia (FA)Phase 1/2ActiveGlobal
- SGT-501SGT-501 / Gene Therapy / CardiacRYR2-Mediated CPVTPhase 1/2ActiveGlobalCASQ2-Mediated CPVTPreclinicalActiveGlobal
- SGT-601SGT-601 / TNNT2 / Gene Therapy / CardiacTNNT2-Mediated Dilated Cardiomyopathy (DCM)PreclinicalActiveGlobal
- SGT-401SGT-401 / BAG3 / Gene Therapy / CardiacBAG3-Mediated Dilated Cardiomyopathy (DCM)PreclinicalActiveGlobal
- SGT-701SGT-701 / RBM20 / Gene Therapy / CardiacRBM20-Mediated Dilated Cardiomyopathy (DCM)PreclinicalActiveGlobal
- Undisclosed Cardiac TargetsGene Therapy / CardiacGenetic cardiomyopathies and channelopathiesDiscoveryActiveGlobal
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- SPY001SPY001 / α4β7 / Monoclonal antibody / GastroenterologyInflammatory Bowel Disease (IBD)Ulcerative Colitis
- SPY002SPY002 / TL1A / Monoclonal antibody / Gastroenterology / RheumatologyInflammatory Bowel Disease (IBD)Rheumatoid Arthritis (RA)
- SPY003SPY003 / IL-23 / Monoclonal antibody / GastroenterologyInflammatory Bowel Disease (IBD)
- SPY120SPY120 / α4β7 + TL1A / Combination / GastroenterologyInflammatory Bowel Disease (IBD)
- SPY130SPY130 / α4β7 + IL-23 / Combination / GastroenterologyInflammatory Bowel Disease (IBD)
- SPY230SPY230 / TL1A + IL-23 / Combination / GastroenterologyInflammatory Bowel Disease (IBD)Ulcerative Colitis
- SPY072SPY072 / TL1A / Monoclonal antibody / RheumatologyRheumatoid Arthritis (RA)Psoriatic Arthritis (PsA)Axial Spondyloarthritis (axSpA)
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- VY7523VY-TAU01 / Tau / Monoclonal Antibody / NeurologyAlzheimer's DiseasePhase 1OngoingUnited States
- VY1706Tau / AAV Gene Therapy / NeurologyAlzheimer's DiseaseIND-EnablingOngoingUnited States
- APOE Gene TherapyAPOE / AAV Gene Therapy / NeurologyAlzheimer's DiseasePreclinicalOngoing
- NBIB-'223NBIB-223 / Frataxin (FXN) / AAV Gene Therapy / NeurologyFriedreich's Ataxia (FA)IND-EnablingOngoing
- GBA1 Gene TherapyGBA1 / AAV Gene Therapy / NeurologyParkinson's Disease and GBA1-mediated diseasesIND-EnablingOngoing
- Novartis Partnered ProgramsUndisclosed / AAV Gene Therapy / NeurologyHuntington's Disease, Spinal Muscular Atrophy (SMA), UndisclosedPreclinicalOngoing
- Alexion Partnered ProgramUndisclosed / AAV Gene Therapy / NeurologyUndisclosed Neurological DiseasePreclinicalOngoing
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- RemlifanserinACP-204 / 5-HT2A receptor / inverse agonist / Neurological DisordersAlzheimer’s Disease PsychosisPhase 2RecruitingLewy Body Dementia with PsychosisPhase 2
- ACP-211ACP-211 / selectively deuterated R-norketamine / Neurological DisordersMajor Depressive DisorderPhase 2
- ACP-711ACP-711 / GABAA α3 / modulator / Neurological DisordersEssential TremorPhase 1
- ACP-271ACP-271 / GPR88 / agonist / Neurological Disorders; Rare DiseasesTardive DyskinesiaIND EnablingHuntington's DiseaseIND Enabling
- ACP-2591ACP-2591 / cGP analog / Rare DiseasesRett Syndrome; Fragile X SyndromePhase 1
- STOKE ASOantisense oligonucleotide / Rare DiseasesSYNGAP1Discovery
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- ACI-35.030JNJ-2056; JNJ-64042056 / phosphorylated Tau (pTau) / active immunotherapy / neurodegenerative diseasespreclinical Alzheimer's diseasePhase 2bongoing
- ACI-24.060amyloid beta (Abeta) / active immunotherapy / neurodegenerative diseasesAlzheimer's diseasePhase 2ongoingAlzheimer's disease in Down syndromePhase 2ongoing
- ACI-7104.056alpha-synuclein (a-syn) / active immunotherapy / neurodegenerative diseasesParkinson's diseasePhase 2ongoing
- ACI-19764NLRP3 inflammasome / small molecule inhibitor / neuroinflammationneuro-inflammationPhase 1ongoing
- Morphomer TauTau / small molecule / neurodegenerative diseasesAlzheimer's diseasePreclinicalIND-enabling studies planned
- Morphomer a-synalpha-synuclein (a-syn) / small molecule / neurodegenerative diseasesParkinson's diseasePreclinicalreadiness to initiate IND-enabling studies
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- AL101PGRN / antibody / Neurodegenerative diseasesAlzheimer’s diseasePhase 2
- AL137-ABCAmyloid beta / ABC-enabled antibody / Neurodegenerative diseasesAlzheimer’s diseasePreclinical
- AL050-ABCGCase / ABC-enabled enzyme replacement therapy / Neurodegenerative diseasesParkinson’s diseasePreclinicalGaucher diseasePreclinicalLewy body dementiaPreclinical
- AL064-ABCTau / ABC-enabled siRNA / Neurodegenerative diseasesAlzheimer’s diseaseResearchFrontotemporal dementiaResearch
- ADP062-ABCAlpha-synuclein / ABC-enabled siRNA / Neurodegenerative diseasesParkinson’s diseaseResearch
- ADP065-ABCNLRP3 / ABC-enabled siRNA / Neurodegenerative diseasesAlzheimer’s diseaseResearchParkinson’s diseaseResearch
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- cemacabtagene ansegedleucelcema-cel; ALLO-501A / CD19 / Allogeneic CAR T cell therapy / Hematologic malignanciesLarge B-cell lymphoma, MRD-positive first-line consolidationPivotal Phase 2EnrollingUSCanadaSouth KoreaAustralia
- ALLO-316ALLO-316 / CD70 / Allogeneic CAR T cell therapy / Solid tumors; hematologic malignanciesAdvanced/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 diseaseSystemic lupus erythematosus including lupus nephritisPhase 1OngoingIdiopathic inflammatory myopathiesPhase 1OngoingSystemic sclerosisPhase 1Ongoing
- ALLO-213ALLO-213 / DLL3 / Allogeneic CAR T cell therapy / Solid tumorsSmall cell lung cancerIND-enabling
- ALLO-182ALLO-182 / Claudin 18.2 / Allogeneic CAR T cell therapy / Solid tumorsGastric and pancreatic solid tumorsIND-enabling
- BCMA/CD70 dual CAR T constructBCMA/CD70 / Allogeneic dual CAR T cell therapy; Dagger technology / Hematologic malignanciesMultiple myelomaPreclinical
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- APL-3007 + SYFOVREAPL-3007 / SYFOVRE / C3 / siRNA + pegcetacoplan injection / OphthalmologyGeographic atrophyPhase 2
- EMPAVELIpegcetacoplan / EMPAVELI / C3 / C3 inhibitor / Rare diseaseFSGSPivotal Phase 2/3DGFPivotal Phase 3
- RNA therapiesRNA therapy / NeurologyUndisclosedPreclinical
- APL-9099: Gene-edited FcRn therapyAPL-9099 / FcRn / gene-edited therapy / Multiple therapeutic areasUndisclosedPreclinical
- Gene-edited complement therapiescomplement / gene-edited therapy / Multiple therapeutic areasUndisclosedPreclinical
- Oral complement inhibitorcomplement / oral complement inhibitor / Multiple therapeutic areasUndisclosedPreclinical
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- obe-celAUCATZYL / CD19 / autologous CAR T-cell therapy / Hematology/oncology; autoimmuner/r adult ALLApprovedFDA/MHRA/EMA approvedUSUKEUPediatric B-ALLPhase 2CATULUSSystemic Lupus ErythematosusPhase 1CARLYSLE
- AUTO1/22CD19 & CD22 / dual-targeting autologous CAR T-cell therapy / Hematology/oncologyPediatric B-ALLPhase 1CARPALL
- AUTO8BCMA & CD19 / autologous CAR T-cell therapy / Hematology/oncologyMultiple MyelomaPhase 1MCARTYLight Chain AmyloidosisPhase 1ALARIC
- AUTO4/5TRBC1/2 / programmed T-cell therapy / Hematology/oncologyTRBC1/2+ Peripheral TCLPreclinicalLibrA T1
- AUTO6NGGD2 / autologous CAR T-cell therapy / Solid tumorsNeuroblastomaPhase 1MAGNETO
- AUTO9programmed T-cell therapy / Hematology/oncologyAcute Myeloid LeukemiaPreclinicalTBD
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- AXS-05AXS-05 / AUVELITY / NMDA receptor; sigma-1 receptor; CYP2D6 / oral small molecule; dextromethorphan-bupropion / CNSSmoking cessationPhase 2/3 plannedOn track to initiate in Q2 2026US
- SolriamfetolSUNOSI / DNRI; TAAR1; 5-HT1A / oral small molecule / CNSADHD, 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 / CNSCataplexy in narcolepsyNDA submittedFDA filing acceptance decision pendingUS
- AXS-14AXS-14 / Norepinephrine reuptake inhibitor / oral small molecule; esreboxetine / CNSFibromyalgiaPhase 3FORWARD ongoingUS
- AXS-17AXS-17 / GABA-A receptor α2,3 subtype-selective PAM / oral small molecule / CNSEpilepsyPhase 2 trial-enablingUnderwayUS
- AXS-20AXS-20 / PDE10A / oral small molecule; balipodect / CNSSchizophreniaPre-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 / HematologySickle 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 / HematologySickle cell diseasePhase I-EnablingBeta-thalassemiaPhase I-Enabling
- In vivo HSC editingHbF activation / In vivo LNP base editing / HematologySickle cell diseaseResearchBeta-thalassemiaResearch
- BEAM-302BEAM-302 / SERPINA1 E342K/PiZ mutation correction / In vivo LNP base editing / Liver-targeted genetic diseasesAlpha-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 diseasesGlycogen storage disease IaPhase I/IIOngoing Phase 1/2 trial
- BEAM-304BEAM-304 / PAH mutation correction / In vivo LNP base editing / Liver-targeted genetic diseasesPhenylketonuriaPhase I-EnablingPlanned Phase 1/2 trialU.S.
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- BXCL501BXCL501 / IGALMI / alpha-2 receptor agonist / orally dissolving/sublingual film formulation of dexmedetomidine / NeuroscienceAcute 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-oncologySmall 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 / NeuroscienceChronic agitation in dementiaPreclinicalDevelopment paused due to strategic reprioritization
- BXCL503BXCL503 / NeuroscienceApathy in dementiaPreclinicalDevelopment paused due to strategic reprioritization
- BXCL504BXCL504 / NeuroscienceAggression in dementiaPreclinicalDevelopment paused due to strategic reprioritization
- BXCL702BXCL702 / DPP inhibitor / novel DPP inhibitor / Immuno-oncologySolid tumorsPreclinicalBXCL701 follow-on; candidate nomination expected
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- Oral Pan-viral protease Inhibitor CDI-988CDI-988 / pan-viral protease / oral small-molecule antiviral / AntiviralNorovirusPhase 1Phase 1b challenge study initiated
- Oral Pan-viral protease Inhibitor CDI-988CDI-988 / pan-viral protease / oral small-molecule antiviral / AntiviralCoronavirusPhase 1Phase 1 study completed
- Pan-viral protease inhibitorpan-viral protease / small-molecule antiviral / AntiviralRhinovirusPreclinicalLead discovery ongoing
- Oral PB2 inhibitor CC-42344CC-42344 / PB2 / oral small-molecule antiviral / AntiviralInfluenza APhase 2Phase 2a study completed, additional Phase 2a needed
- Inhaled PB2 inhibitor CC-42344CC-42344 / PB2 / inhaled small-molecule antiviral / AntiviralInfluenza APhase 3GLP tox study complete
- Oral replication inhibitorviral replication / oral small-molecule antiviral / AntiviralInfluenza A & BPreclinicalLead discovery ongoing, NIH SBIR Funded
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- AmglidiaAmglidia / glyburide oral suspension / Pediatric endocrinology / rare diseaseNeonatal diabetes mellitusLate-stage development; NDA submission anticipated in 2026In developmentUnited States
- ET-700ET-700 / extended-release zinc acetate formulation / Rare metabolic diseaseWilson diseasePilot clinical study / proof-of-conceptFirst patient dosed; topline results expected H2 2026; potential pivotal study in early 2027United States
- ET-800ET-800 / Rare diseaseUndisclosedLate-stage developmentIn development
- ZENEO hydrocortisone autoinjectorZENEO / hydrocortisone autoinjector / EndocrinologyAdrenal insufficiency / adrenal crisis managementLate-stage developmentIn developmentUnited States
- KHINDIVI revised formulationKHINDIVI / hydrocortisone oral solution / Pediatric endocrinologyAdrenocortical 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 endocrinologySevere 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 / AutoimmunitySystemic 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; OncologyPan-indication without lympho-conditioning chemotherapyPhase 1USPan-indication (hematologic) without lympho-conditioning chemotherapyPhase 1US
- FT522FT522 / CD19/CD20 / Cellular immunotherapy / AutoimmunityPan-indication without lympho-conditioning chemotherapyPhase 1US
- FT825FT825 / HER2/EGFR / iPSC-derived off-the-shelf CAR T-cell therapy / OncologySolid tumorsPhase 1EnrollingUS
- UndisclosedUndisclosed / Cellular immunotherapy / OncologySolid tumorsPhase 1US
- FT836FT836 / MICA/B/EGFR/HER2 / Next-generation CAR T-cell therapy / OncologyPan-indication (solid/hematologic) without lympho-conditioning chemotherapyPhase 1US
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- Symvess (ATEV)Symvess / Acellular Tissue Engineered Vessel / Vascular DiseaseVascular 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 DiseaseCoronary artery bypass grafting (CABG)PreclinicalPositive NHP data; IND submitted; first-in-human planned H2 2026United States
- Biovascular Pancreas (BVP)Biovascular Pancreas™ / Bioengineered Organ / DiabetesType 1 diabetesPreclinicalPositive preclinical data; NHP studies planned; first-in-human planned 2027
- Urinary ConduitAdvanced Tissue Construct / UrologyUrinary diversion after cystectomyPreclinical
- Tracheal ReplacementAdvanced Tissue Construct / OtolaryngologyTracheal replacementPreclinical
- Esophageal ReplacementAdvanced Tissue Construct / GastroenterologyEsophageal replacementPreclinical
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- INO-3107INO-3107 / HPV-6 and HPV-11 / DNA immunotherapy / HPV-related rare diseaseRecurrent Respiratory Papillomatosis (RRP) in adultsBLA accepted under accelerated approval, PDUFA Oct 30 2026United States
- VGX-3100VGX-3100 / HPV-16/18 / DNA immunotherapy / HPV-related diseasesCervical dysplasia (HSIL)3Ongoing (partnered)ChinaAnal dysplasia
- INO-3112INO-3112 / HPV-16/18 / DNA immunotherapy / OncologyHPV16/18-positive oropharyngeal squamous cell carcinoma (OPSCC)3Phase 3 planningUnited StatesEurope
- INO-5401INO-5401 / Tumor-associated antigens / DNA immunotherapy / OncologyGlioblastoma (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 diseasesEbola vaccine booster (to ERVEBO)2Phase 2 planning; Phase 1b completed
- DMAb SARS-CoV-2SARS-CoV-2 / DNA-encoded monoclonal antibody / Infectious diseasesSARS-CoV-21Ongoing Phase 1; data expected/reportedUnited States
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- PemivibartPEMGARDA / SARS-CoV-2 / Monoclonal Antibody / Infectious DiseasesPre-exposure prophylaxis (PrEP) of COVID-19
- VYD2311VYD2311 / SARS-CoV-2 / Monoclonal Antibody / Infectious DiseasesCOVID-19 prevention
- AdintrevimabSARS-CoV-2 / Monoclonal Antibody / Infectious DiseasesCOVID-19 prevention and treatment
- Measles mAbMeasles (rubeola) virus / Monoclonal Antibody / Infectious DiseasesMeasles treatment and post-exposure prophylaxis
- RSV mAbRespiratory Syncytial Virus (RSV) / Monoclonal Antibody / Infectious DiseasesRSV infection
- Influenza mAbInfluenza virus / Monoclonal Antibody / Infectious DiseasesInfluenza infection
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- MP0712MP0712 / DLL3 / Radio-DARPin Therapy / OncologySmall Cell Lung Cancer (SCLC)Neuroendocrine Tumors
- MP0726MP0726 / MSLN / Radio-DARPin Therapy / OncologyOvarian Cancer
- MP0533MP0533 / CD33 x CD123 x CD70 x CD3 / Multispecific T Cell Engager / OncologyRelapsed/Refractory AMLAML/MDS
- MP0317MP0317 / FAP x CD40 / Tumor-localized CD40 Agonist / OncologyAdvanced Solid TumorsCholangiocarcinoma
- MP0632MP0632 / CD2 x CD3 x MSLN x EpCAM / Switch-DARPin T Cell Engager / OncologyMSLN/EpCAM Expressing Solid TumorsOvarian Cancer
- MP0621MP0621 / cKIT x CD16a x CD47 / Switch-DARPin / HematologyConditioning 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 DiseasesObesityPhase 1ActiveGlobalMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 1ActiveGlobal
- VanoglipelDA-1241 / GPR119 (G-protein-coupled receptor 119) / Small Molecule / Cardiometabolic DiseasesMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 2aActiveGlobalType 2 Diabetes (T2D)Phase 1CompletedGlobal
- ANA001ANA001 / Unknown / Oral Niclosamide Formulation / Infectious DiseasesModerate COVID-19Phase 2LegacyUS
- NB-01NB-01 / Unknown / Unknown / NeurologyPainful Diabetic NeuropathyUnknownLegacyGlobal
- NB-02NB-02 / Unknown / Unknown / NeurologyCognitive Impairment / Neurodegenerative DiseasesUnknownLegacyGlobal
- GemcabeneUnknown / Small Molecule / Cardiovascular / Infectious DiseasesDyslipidemiaUnknownLegacyGlobalAcute COVID-19UnknownLegacyGlobal
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- ebopiprantPreterm labor
- OG-6219Endometriosis
- OG-7191PCOS
- Cirqle Biomedical candidateNon-hormonal contraceptive
- Xaciatoclindamycin phosphate vaginal gel
- Jada systemPostpartum hemorrhage device
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- OST-HER2OST31-164 / HER2 / Bioengineered Listeria monocytogenes (Immunotherapy) / OncologyOsteosarcoma (Pulmonary Metastatic)Phase 2bActiveGlobalBreast CancerPhase 1ActiveGlobalCanine OsteosarcomaApprovedMarketedUS
- OST-AXALAXAL / ADXS-HPV / HPV / Listeria-based Immunotherapy / OncologyHPV-associated cancersPhase 3ActiveGlobal
- OST-503ADXS-503 / KRAS G12 position-related antigens / Listeria-based Immunotherapy / OncologyNon-Small Cell Lung Cancer (NSCLC)Phase 2ActiveGlobalGlioblastoma (GBM)Phase 2ActiveGlobalPancreatic CancerPreclinicalActiveGlobal
- OST-504OST-PSA / ADXS-504 / ADXS31142 / PSA / Listeria-based Immunotherapy / OncologyProstate Cancer (Castration-Resistant)Phase 1bActiveGlobal
- OST-tADCVarious / Tunable Antibody-Drug Conjugate (ADC) / OncologyOvarian CancerPreclinicalActiveGlobalBreast CancerPreclinicalActiveGlobal
- OST-HOT constructsShared hotspot mutations and cancer-testes antigens / Listeria-based Immunotherapy / OncologySolid TumorsPreclinicalActiveGlobal
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- AX-0810AX-0810 / NTCP (SLC10A1) / RNA Editing Oligonucleotide (Axiomer EON) / LiverBiliary AtresiaPrimary Sclerosing Cholangitis
- AX-0811AX-0811 / NTCP / RNA Editing Oligonucleotide (Axiomer EON) / LiverCholestatic Diseases
- AX-0422AX-0422 / IDUA / RNA Editing Oligonucleotide (Axiomer EON) / Metabolic / Rare DiseaseHurler Syndrome
- AX-2402AX-2402 / MECP2 (R270X) / RNA Editing Oligonucleotide (Axiomer EON) / CNS / Rare DiseaseRett Syndrome
- AX-2911AX-2911 / PNPLA3 / RNA Editing Oligonucleotide (Axiomer EON) / Liver / MetabolicMASH
- AX-1412AX-1412 / B4GALT1 / RNA Editing Oligonucleotide (Axiomer EON) / CardiovascularCardiovascular Disease
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- rivunatpagene miziparvovecUX701 / ATP7B / AAV9 gene therapy / Inborn Errors of MetabolismWilson disease
- rebisufligene etisparvovecUX111 / AAV gene therapy / NeurogeneticSanfilippo syndrome type A (MPS IIIA)
- avalotcagene ontaparvovecDTX301 / OTC / AAV8 gene therapy / Inborn Errors of MetabolismOrnithine transcarbamylase (OTC) deficiency
- DTX401DTX401 / AAV gene therapy / Inborn Errors of MetabolismGlycogen storage disease type Ia (GSDIa)
- GTX-102GTX-102 / UBE3A / Antisense oligonucleotide (ASO) / NeurogeneticAngelman Syndrome
- setrusumabUX143 / Biologic / Bone-EndocrineOsteogenesis imperfecta
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- DaraxonrasibRMC-6236 / RAS(ON) multi-selective / Small Molecule / OncologyPancreatic Ductal Adenocarcinoma (PDAC)Non-Small Cell Lung Cancer (NSCLC)Colorectal Cancer (CRC)
- ZoldonrasibRMC-9805 / RAS(ON) G12D / Small Molecule / OncologyNon-Small Cell Lung Cancer (NSCLC)Solid Tumors
- ElironrasibRMC-6291 / RAS(ON) G12C / Small Molecule / OncologyKRAS-G12C mutant cancersNon-Small Cell Lung Cancer (NSCLC)Solid Tumors
- RMC-5127RMC-5127 / RAS(ON) G12V / Small Molecule / OncologyRAS-G12V mutant cancers
- RMC-0708RMC-0708 / RAS(ON) Q61H / Small Molecule / OncologyRAS-Q61H mutant cancers
- RMC-8839RMC-8839 / RAS(ON) G13C / Small Molecule / OncologyRAS-G13C mutant cancers
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- SGR-1505SGR-1505 / MALT1 / Small Molecule / OncologyRelapsed/Refractory B-cell MalignanciesPhase 1Active
- SGR-3515SGR-3515 / PKMYT1 / Wee1 / Small Molecule / OncologyAdvanced Solid TumorsPhase 1Active
- SGR-4174SGR-4174 / SOS1 / Small Molecule / OncologyKRAS-driven CancersPreclinicalActive
- SGR-2921SGR-2921 / CDC7 / Small Molecule / OncologyAcute Myeloid Leukemia (AML) / Myelodysplastic Syndrome (MDS)Phase 1Discontinued
- ACCG-2671ACCG-2671 / Calcitonin Receptor (DACRA) / Small Molecule / MetabolicObesityPhase 1Active
- AJ1-11095AJ1-11095 / JAK2 / Small Molecule / HematologyMyelofibrosisPreclinicalActiveUS
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- DivesiranSLN124 / TMPRSS6 / siRNA / Hematologic ConditionsPolycythemia VeraOther Blood Disorders
- ZerlasiranSLN360 / LPA / siRNA / Cardiovascular DiseaseElevated Lp(a)Atherosclerotic Cardiovascular Disease
- SLN312SLN312 / ANGPTL3 / siRNA / CardiometabolicDyslipidemia
- SLN548SLN548 / CFB / siRNA / Complement
- SLN365SLN365 / GPR146 / siRNA / CardiometabolicHomozygous familial hypercholesterolemia (HoFH)
- SLN098SLN098 / INHBE / siRNA / MetabolicObesity
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- AmezalpatTPST-1120 / PPARα antagonist / Small Molecule / OncologyHepatocellular Carcinoma (HCC)CholangiocarcinomaRenal Cell Carcinoma (RCC)
- TPST-1495TPST-1495 / Dual EP2/EP4 Antagonist / Small Molecule / Oncology / GastrointestinalFamilial Adenomatous Polyposis (FAP)Solid Tumors
- TREX-1 InhibitorTREX-1 / Small Molecule / OncologySolid Tumors
- TPST-2003TPST-2003 / CD19/BCMA / Autologous CAR-T Cell Therapy / OncologyRelapsed/Refractory Multiple Myeloma (rrMM)
- TPST-3003TPST-3003 / CD19/BCMA / Allogeneic Cell Therapy / OncologyRelapsed/Refractory Multiple Myeloma (rrMM)
- TPST-4003TPST-4003 / CD19/BCMA / In Vivo Cell Therapy / Immunology / AutoimmuneSystemic Lupus Erythematosus (SLE)Immunology Disorders
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- TreprostinilTyvaso / Prostacyclin receptor / Small Molecule / RespiratoryIdiopathic Pulmonary Fibrosis (IPF)
- RalinepagProstacyclin receptor / Small Molecule / CardiopulmonaryPulmonary Arterial Hypertension (PAH)
- UKidneyUKidney / Xenotransplantation / Organ TransplantationEnd Stage Renal Disease
- UThymoKidneyUThymoKidney / Xenotransplantation / Organ TransplantationEnd Stage Renal Disease
- miroliverELAPmiroliverELAP / Regenerative Medicine / Organ TransplantationAcute Liver Failure
- Centralized Lung Evaluation System (CLES)Ex-vivo Perfusion System / Organ TransplantationLung Transplant
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- Chikungunya vaccineVLA1553 / IXCHIQ
- Lyme Disease vaccineVLA15
- Shigella vaccineS4V2
- Zika vaccineVLA1601
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- ElebsiranVIR-2218 / HBV / siRNA / Infectious DiseasesChronic Hepatitis Delta (CHD)Phase 3ActiveGlobalChronic Hepatitis B (CHB)Phase 2ActiveGlobal
- TobevibartVIR-3434 / HBV / Monoclonal Antibody / Infectious DiseasesChronic Hepatitis Delta (CHD)Phase 3ActiveGlobalChronic Hepatitis B (CHB)Phase 2ActiveGlobal
- VIR-1388VIR-1388 / HIV / T Cell Vaccine / Infectious DiseasesHIV PreventionPhase 1ActiveUnited StatesSouth Africa
- VIR-5500VIR-5500 / PSMA / Masked T-Cell Engager / OncologySolid TumorsPhase 1ActiveGlobal
- VIR-5525VIR-5525 / EGFR / Masked T-Cell Engager / OncologySolid TumorsPhase 1ActiveGlobal
- VIR-5818VIR-5818 / HER2 / Masked T-Cell Engager / OncologySolid TumorsPhase 1ActiveGlobal
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- VK2735 (Subcutaneous)VK2735 / Dual GLP-1/GIP receptor agonist / Peptide / Metabolic DisordersObesity
- VK2735 (Oral)VK2735 / Dual GLP-1/GIP receptor agonist / Peptide / Metabolic DisordersObesity
- VK2809VK2809 / Thyroid hormone receptor beta (TRβ) agonist / Small molecule / Metabolic DisordersNASHFibrosisNAFLD
- VK0214VK0214 / Thyroid hormone receptor beta (TRβ) agonist / Small molecule / Rare DiseasesX-linked adrenoleukodystrophy (X-ALD)
- Amylin Agonist (DACRAs)Dual amylin and calcitonin receptor agonist (DACRA) / Agonist / Metabolic DisordersObesityMetabolic disorders
- VK5211VK5211 / Selective androgen receptor modulator (SARM) / Small molecule / Musculoskeletal DisordersHip fracture recoveryMuscle and bone disorders
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- fasedienolPH94B / peripheral nasal chemosensory receptors / Intranasal pherine / PsychiatrySocial Anxiety Disorder (SAD)
- itruvonePH10 / peripheral nasal chemosensory receptors / Intranasal pherine / PsychiatryMajor Depressive Disorder (MDD)
- refisolonePH80 / peripheral nasal chemosensory receptors / Intranasal pherine / Women's HealthMenopausal Vasomotor SymptomsPremenstrual Dysphoric Disorder (PMDD)
- PH15PH15 / peripheral nasal chemosensory receptors / Intranasal pherine / Neurology/PsychiatryPsychomotor and cognitive impairment due to mental fatigue
- PH284PH284 / peripheral nasal chemosensory receptors / Intranasal pherine / Oncology Supportive CareCancer Cachexia
- AV-101AV-101 / NMDA receptor glycine site / Small molecule / Neurology/PsychiatryNeurological disorders
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- VilastobartXTX101 / CTLA-4 / Monoclonal Antibody / OncologyAdvanced solid tumors / MSS mCRCPhase 2ActiveGlobal
- Efarindodekin alfaXTX301 / IL-12 / Cytokine / OncologyAdvanced solid tumorsPhase 2ActiveGlobal
- XTX501XTX501 / PD-1 / IL-2 / Bispecific Antibody / Cytokine / OncologyNon-small cell lung cancer (NSCLC) and other solid tumorsPreclinicalActiveGlobal
- PSMA+STEAP1 Masked T Cell EngagerPSMA / STEAP1 / T Cell Engager / OncologyProstate cancerPreclinicalActiveGlobal
- CLDN18.2 Masked T Cell EngagerCLDN18.2 / T Cell Engager / OncologyGastric, pancreatic, esophageal, and lung cancersPreclinicalActiveGlobal
- Undisclosed Masked T Cell EngagerUndisclosed / T Cell Engager / OncologyCancerPreclinicalActiveGlobal
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- BosakitugATI-045 / TSLP / humanized anti-TSLP monoclonal antibody / immuno-inflammatory; respiratory; dermatologyatopic dermatitisPhase 2ongoing
- ATI-052ATI-052 / TSLP; IL-4Rα / humanized bispecific antibody / immuno-inflammatory; respiratory; dermatologyatopic dermatitisPhase 1bongoingasthmaPhase 1bongoingasthmaPhase 2bplanned
- ATI-2138ATI-2138 / ITK; JAK3 / oral covalent small-molecule inhibitor / T cell-mediated autoimmune diseases; dermatologylichen planusPhase 2bplannedatopic dermatitisPhase 2acompleted
- ATI-9494ATI-9494 / ITK; TXK/RLK / oral covalent small-molecule inhibitor / Th1, Th2 and Th17-driven diseasesundisclosed inflammatory diseasespreclinicalIND planned
- LepzacitinibATI-1777 / JAK1; JAK3 / topical soft small-molecule inhibitor / dermatologyatopic dermatitisPhase 2bseeking partner
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- TanruprubartANX005 / C1q / classical complement pathway / IV mAb / Autoimmune / neurologyGuillain-Barré SyndromeMAA filed; FDA discussions ongoingactiveUSEU
- VonaprumentANX007 / C1q / classical complement pathway / IVT Fab / OphthalmologyGeographic AtrophyPhase 3ARCHER II enrollment completed; data expected 2H26
- ANX1502ANX1502 / Classical complement pathway / Oral small molecule / AutoimmuneCold Agglutinin DiseaseProof-of-conceptactive; update upon CAD study completion in 2026
- ANX005ANX005 / C1q / classical complement pathway / IV mAb / NeurodegenerationHuntington’s DiseasePoised for late-stage Phase 2b/3activeAmyotrophic Lateral SclerosisPoised for late-stage Phase 2b/3active
- ANX009ANX009 / C1q / classical complement pathway / Subcutaneous Fab / AutoimmuneLupus NephritisEvaluating options for future development
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- TransCon hGHSKYTROFA / growth hormone / TransCon prodrug therapy / Endocrinology Rare DiseasesTurner 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 DiseasesAchondroplasia (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 DiseasesAchondroplasia (children aged 2–11)Phase 2Multinational
- TransCon PTHYORVIPATH / parathyroid hormone / TransCon prodrug therapy / Endocrinology Rare DiseasesAdult Hypoparathyroidism (dosing expansion)Phase 3United StatesAdult HypoparathyroidismPhase 3China
- TransCon IL-2 β/γ (Systemic)IL-2 β/γ / TransCon prodrug therapy / OncologyVarious tumor typesPhase 2Multinational
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- FirmonertinibEGFR TKI / oral small molecule / Oncology1L NSCLC EGFR exon 20 insertion mutationsPhase 3Monotherapyglobal1L+ NSCLC EGFR PACC mutationsPhase 3MonotherapyglobalAdjuvant EGFR uncommon mutationsPhase 3Monotherapy
- ARR-217ARR-217 / CDH17 ADC / antibody-drug conjugate / OncologyGI tumorsPreclinical
- ARR-002ARR-002 / antibody-drug conjugate / OncologySolid tumorsDiscovery
- ARR-421ARR-421 / antibody-drug conjugate / OncologySolid tumorsDiscovery
- ARR-173ARR-173 / antibody-drug conjugate / OncologySolid tumorsDiscovery
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- AcoramidisAttruby / TTR (Transthyretin) / Small Molecule / Genetic diseaseEarly-Stage Variant Transthyretin Amyloidosis (ATTRv)Phase 3Under investigation
- BBP-418BBP-418 / FKRP / Small Molecule / Genetic disease; neuromuscularLimb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)Phase 3Investigational; FORTIFY interim analysis met endpoints
- EncaleretCASR / Small Molecule / Endocrine; genetic diseaseAutosomal 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 diseaseAchondroplasiaPhase 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; neurologyCanavan DiseaseInvestigational AAV gene therapy
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- ORLADEYO (berotralstat)ORLADEYO / plasma kallikrein / oral small-molecule inhibitor / Rare diseasesHereditary angioedema (HAE) prophylaxisApproved/Commercialmarketed
- ORLADEYO (berotralstat) in PediatricsORLADEYO / plasma kallikrein / oral small-molecule inhibitor / Rare diseasesHereditary angioedema (HAE) prophylaxis, ages 2 to <12Approved/Commercialmarketed
- NavenibartSTAR-0215 / plasma kallikrein / monoclonal antibody inhibitor / Rare diseasesHereditary angioedema (HAE) prophylaxisPhase 3/Pivotalinvestigational
- BCX17725BCX17725 / KLK5 / protein therapeutic / Rare diseasesNetherton syndromePhase 1investigational
- STAR-0310STAR-0310 / OX40 / monoclonal antibody antagonist / Immunology/DermatologyAtopic dermatitisPreclinicalinvestigational; strategic alternatives planned
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- nuzefatide pevedotinBT5528 / EphA2 / Bicycle Drug Conjugate / OncologyHigh EphA2-expressing tumorsPhase I/IIOngoingMetastatic pancreatic ductal adenocarcinomaPhase IIOngoing
- zelenectide pevedotinBT8009 / Nectin-4 / Bicycle Drug Conjugate / OncologyNectin-4-expressing tumorsPhase I/IIOngoing Duravelo-1Metastatic urothelial cancerPhase IIOngoing Duravelo-2
- BT7480BT7480 / Nectin-4/CD137 / Bicycle TICA / OncologyAdvanced solid tumors associated with Nectin-4 expressionPhase I/IIOngoing; seeking partner for future development
- BT1702BT1702 / MT1-MMP / Bicycle Radioligand / OncologyMT1-MMP-expressing tumorsIND-enabling / human imagingCompany-sponsored BRC clinical trial planned in 2027
- ION286/AZD4063ION286/AZD4063 / TfR1 / TfR1-targeted Bicycle conjugate / CardiomyopathyCardiomyopathyPhase 1Ongoing
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- BMN 333BMN 333 / C-type natriuretic peptide pathway / Long-acting CNP peptide / Skeletal ConditionsAchondroplasiaPhase 2/3Registration-enabling study enrolling
- BMN 351BMN 351 / Dystrophin / Oligonucleotide / InnovationDuchenne muscular dystrophyPhase 1/2Clinical data update expected year-end 2026
- BMN 401BMN 401 / ENPP1 / Subcutaneous enzyme replacement therapy / Enzyme TherapiesENPP1 deficiencyPhase 3ENERGY 3 met 1 of 2 co-primary endpoints; next steps under evaluation
- vosoritideVOXZOGO / CNP receptor/NPR-B pathway / Peptide / Skeletal ConditionsHypochondroplasiaPhase 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 TherapiesPhenylketonuria adolescents 12+RegulatoryFDA approved February 2026; EU approval expected 2026USEU
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- BDC-4182BDC-4182 / Claudin 18.2 / Boltbody ISAC / OncologyGastric cancerPhase 1Ongoing dose-escalationGastroesophageal cancerPhase 1Ongoing dose-escalation
- BDC-3042BDC-3042 / Dectin-2 / Agonist monoclonal antibody / OncologyNon-small cell lung cancer, melanoma and other solid tumorsPhase 1Dose-escalation complete; seeking partner
- CEA ISACCEA/CEACAM5 / Boltbody ISAC / OncologyColorectal, non-small cell lung, pancreatic and gastric cancersPreclinicalPre-IND; seeking partner
- PD-L1 ISACPD-L1 / Boltbody ISAC / OncologySolid tumors resistant to checkpoint inhibitorsPreclinicalPre-IND; seeking partner
- Caprin-1 ISACCaprin-1 / Boltbody ISAC / Oncology
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- Lasme-celCD22 / allogeneic CAR-T / Oncologyrelapsed/refractory B-cell acute lymphoblastic leukemiaPhase 2BALLI-01; pivotal Phase 2 first interim analysis expected Q4 2026
- Eti-celCD20, CD22 / dual allogeneic CAR-T / Oncologyrelapsed/refractory Non-Hodgkin lymphomaPhase 1NatHaLi-01; full Phase 1 dataset expected Q4 2026
- Cemacabtagene ansegedleucelCema-cel / CD19 / allogeneic CAR-T / Oncologylarge B-cell lymphomaPivotal Phase 2ALPHA3
- ALLO-316ALLO-316 / CD70 / allogeneic CAR-T / Oncologyadvanced or metastatic clear cell renal cell carcinomaPhase 1TRAVERSE
- IOV-4001IOV-4001 / PD-1 / TALEN-modified tumor-infiltrating lymphocyte therapy / Oncologyunresectable or metastatic melanomaIOV-GM1-201stage III or IV NSCLCIOV-GM1-201
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- CLS-AX (axitinib)CLS-AX / VEGF receptors-1, -2 and -3 / Small molecule tyrosine kinase inhibitor / OphthalmologyWet AMDPhase 3 readyFDA end-of-Phase 2 alignment
- XIPERE (triamcinolone acetonide injectable suspension)XIPERE; ARCATUS / Corticosteroid injectable suspension / OphthalmologyUveitic macular edemaApproved/commercialUnited StatesAustraliaSingaporeUveitic macular edemaClinical development/commercialization partnerChinaDiabetic macular edemaClinical development/commercialization partnerAsia Pacific ex-Japan
- Bel-SarViral-like drug conjugate / Ophthalmology/oncologyChoroidal melanomaPartner clinical development program
- ABBV-RGX-314ABBV-RGX-314 / anti-VEGF / AAV gene therapy / OphthalmologyDiabetic retinopathyPartner clinical development programWet AMDPartner clinical development program
- AvoralstatPlasma kallikrein / Small molecule plasma kallikrein inhibitor / OphthalmologyDiabetic macular edemaPartner clinical development program
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- bezuclastinibCGT9486 / KIT exon 17/KIT D816V / small molecule tyrosine kinase inhibitor / Oncology; hematologyNonAdvanced 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 / OncologyAdvanced solid tumors with HER2 mutations or HER2 overexpressionPhase 1dose escalation ongoing
- CGT6297CGT6297 / PI3Kα / small molecule inhibitor / OncologyPI3Kα-mutant cancersPhase 1initiated
- CGT1815CGT1815 / pan-KRAS(ON) / small molecule inhibitor / Oncology
- CGT1145CGT1145 / JAK2 V617F / small molecule inhibitor / Hematology
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- 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 diseasesAmyotrophic 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 diseasesAlzheimer's diseaseIND-enablingactive
- COYA 303COYA 303 / IL-2; GLP-1 receptor / COYA 301 + GLP-1 receptor agonist combination / Inflammatory diseasesUndisclosedDiscoveryactive
- COYA 201COYA 201 / Allogeneic Treg-derived exosomes / UndisclosedUndisclosedPreclinicalactive
- COYA 206COYA 206 / Antigen-directed allogeneic Treg-derived exosomes / UndisclosedUndisclosedPreclinicalactive
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- EB613EB613 / PTH 1-34, teriparatide / oral peptide tablet / OsteoporosisPostmenopausal 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 / HypoparathyroidismHypoparathyroidismPreclinicalDevelopment ongoing; pre-IND initiatives underway
- OXMOPK-88006 / GLP-1 & glucagon agonist / oral oxyntomodulin peptide tablet / Obesity / metabolic diseasesObesity and metabolic diseasesPreclinicalIND filing expected late H1 2026U.S.
- GLP-2Long-acting GLP-2 / oral GLP-2 peptide tablet / Short bowel syndromeShort bowel syndromePreclinicalProof-of-concept single-dose pharmacokinetic study completed in rodents
- EB613EB613 / PTH 1-34, teriparatide / oral peptide tablet / Stress fracturesStress fracturesInvestigator Sponsored Trial
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- HydronidoneF351 / TGFβRI/p38γ (anti-fibrotic) / Small molecule / FibrosisCHB-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/RespiratoryIdiopathic 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 / HepatologyAcute/acute-on-chronic liver failure (ALF/ACLF)ClinicalIn developmentChina
- F528F528 / Small molecule / RespiratoryChronic obstructive pulmonary disease (COPD)PreclinicalIn developmentChina
- F230F230 / Small molecule / CardiopulmonaryPulmonary arterial hypertension (PAH)PreclinicalIn developmentChina
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- CrofelemerMytesi / CFTR and CaCC channels / Botanical Drug / GastrointestinalHIV-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 DiseaseMicrovillus Inclusion Disease (MVID)Late StageActiveUnited StatesEuropeShort Bowel Syndrome with Intestinal Failure (SBS-IF)Late StageActiveUnited StatesEurope
- NP-300NP-300 / Unknown / Botanical Drug / Gastrointestinal / Infectious DiseaseCholera Diarrhea / Infectious DiarrheaIn DevelopmentActiveGlobal
- GelclairGelclair / Unknown / Bioadherent Oral Gel / Supportive Care / OncologyOral MucositisApprovedMarketedUnited States
- Crofelemer (Animal Health)Canalevia-CA1 / CFTR and CaCC channels / Botanical Drug / Animal HealthChemotherapy-induced diarrhea in dogsApprovedMarketedUnited States
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- KRRO-121KRRO-121 / Glutamine Synthetase (GS) / GalNAc-conjugated oligonucleotide (RNA Editing) / Metabolic / LiverUrea Cycle Disorders (UCD) / Hepatic Encephalopathy (HE)PreclinicalActiveGlobal
- AATD GalNAc ProgramSERPINA1 mRNA / GalNAc-conjugated oligonucleotide (RNA Editing) / Rare Diseases / Respiratory / LiverAlpha-1 Antitrypsin Deficiency (AATD)PreclinicalActiveGlobal
- AMPKγ1 ProgramAMPKγ1 / GalNAc-conjugated oligonucleotide (RNA Editing) / Metabolic / LiverLiver Function / LongevityPreclinicalActiveGlobal
- ALS ProgramTDP-43 mRNA / Oligonucleotide (RNA Editing) / NeurologyAmyotrophic Lateral Sclerosis (ALS)PreclinicalActiveGlobal
- Cardiometabolic ProgramUndisclosed / Oligonucleotide (RNA Editing) / Metabolic / CardiovascularCardiometabolic DiseasePreclinicalActiveGlobal
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- ziftomenibKOMZIFTI / Menin / Small Molecule / OncologyAcute Myeloid Leukemia (AML) (Combination)Phase 3ActiveGlobalRelapsed or refractory (R/R) NPM1-mutant AMLPre-RegistrationNDA Submitted / Priority ReviewUS
- rocatinlimabKHK4083 / AMG 451 / OX40 / Monoclonal Antibody / Immunology/AllergyModerate to Severe Atopic DermatitisPhase 3ActiveGlobalUSEUJPPrurigo NodularisPhase 3ActiveGlobalAsthmaPhase 2ActiveGlobal
- atidarsagene autotemcelOTL-200 / Lenmeldy / Libmeldy / ARSA / Hematopoietic Stem Cell (HSC) Gene Therapy / Rare DiseasesEarly-onset Metachromatic Leukodystrophy (MLD)Pre-RegistrationPreparation underway for clinical trial / Regulatory ReviewJP
- tivozanibKHK4951 / VEGF Receptor Tyrosine Kinase / Small Molecule / OphthalmicOphthalmic DiseasesPhase 2/3ActiveGlobal
- mogamulizumabKW-0761 / POTELIGEO / CCR4 / Monoclonal Antibody / OncologyMycosis fungoides and Sézary syndromePre-RegistrationFiledCanada
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- sparsentansparsentan / Filspari / ETA/AT1 receptors / Small molecule / NephrologyIgA Nephropathy
- ensifentrineensifentrine / Ohtuvayre / PDE3/PDE4 / Small molecule / RespiratoryCOPD
- pneumococcal 15-valent conjugate vaccineV114 / Vaxneuvance / Pneumococcal serotypes / Vaccine / Infectious DiseasePneumococcal disease
- carfilzomibcarfilzomib / Kyprolis / Proteasome / Small molecule / OncologyMultiple Myeloma
- berdazimerberdazimer / Zelsuvmi / Nitric oxide / Small molecule / DermatologyMolluscum contagiosum
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- CertepetideLSTA1 / alpha-v integrins / Cyclic Peptide / OncologyAdvanced Solid TumorsPancreatic Ductal Adenocarcinoma (PDAC)Cholangiocarcinoma (CCA)
- LSTA12LSTA12 / HONEDRA / CD34+ / Cell Therapy / CardiovascularCritical Limb Ischemia (CLI)
- LSTA14LSTA14 / OLOGO / CD34+ / Cell Therapy / CardiovascularNo-Option Refractory Disabling Angina (NORDA)
- XOWNAXOWNA / CD34+ / Cell Therapy / CardiovascularCoronary Microvascular Dysfunction (CMD)
- LSTA201LSTA201 / CD34+ / Cell Therapy / NephrologyDiabetic Kidney Disease (DKD)
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- LP-300LP-300 / PTPN11 / Small molecule / OncologyNon-small cell lung cancer (NSCLC) in never-smokers
- LP-184LP-184 / PTGR1-activated alkylating agent / Small molecule / OncologySolid tumorsCNS cancersGlioblastoma multiforme (GBM)
- LP-284LP-284 / PKC-beta / Small molecule / OncologyHematologic malignanciesAggressive B-cell lymphomas
- STAR-001STAR-001 / Unknown / Small molecule / OncologyCNS cancersBrain metastases
- ADC ProgramUnknown / Antibody-drug conjugate (ADC) / OncologyCancer
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- CanvuparatideMBX 2109
- ImapextideMBX 1416
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- MGC026B7-H3 ADC
- MGC028ADAM9 ADC
- MGC030TOP1i-based ADC
- lorigerlimabPD-1 x CTLA-4 bispecific DART
- MGD024CD123 x CD3 DART
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- MaralixibatLIVMARLI / IBAT / Small molecule / Rare Liver DiseasesAlagille syndrome (ALGS)Progressive familial intrahepatic cholestasis (PFIC)Biliary atresia
- VolixibatSHP626 / IBAT / Small molecule / Rare Liver DiseasesPrimary sclerosing cholangitis (PSC)Primary biliary cholangitis (PBC)
- MRM-3379MRM-3379 / PDE4D / Small molecule / Neurocognitive DisordersFragile X syndrome
- Cholic acidCHOLBAM / Bile acid replacement / Small molecule / Rare Liver DiseasesBile acid synthesis disordersPeroxisomal disorders (Zellweger spectrum disorders)
- ChenodiolCTEXLI / Bile acid replacement / Small molecule / Rare Genetic DisordersCerebrotendinous xanthomatosis (CTX)
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- ALXN1840ALXN1840 / Copper / Small Molecule / Rare DiseasesWilson Disease
- MNPR-101-ZrMNPR-101-Zr / uPAR / Radiopharmaceutical / OncologyAdvanced Solid Cancers (Imaging)
- MNPR-101-LuMNPR-101-Lu / uPAR / Radiopharmaceutical / OncologyAdvanced Solid Cancers
- MNPR-101-AcMNPR-101-Ac / uPAR / Radiopharmaceutical / OncologyAdvanced Solid Cancers
- MNPR-101MNPR-101 / uPAR / Monoclonal Antibody / OncologySolid Tumors
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- PetosemtamabMCLA-158 / EGFR x LGR5 / Bispecific Antibody / Oncology1L 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 / OncologyPancreatic 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 / OncologySolid tumorsPhase 1/Phase 2OngoingGlobal2L+ EGFRm non-small cell lung cancer (with chemotherapy)Phase 2OngoingGlobal
- BesufetamigONO-4685 / PD-1 x CD3 / Bispecific Antibody / Oncology, ImmunologyRelapsed/Refractory T Cell LymphomaPhase 1OngoingGlobalPsoriasisPhase 1OngoingGlobal
- INCA33890INCA33890 / PD-1 x TGFBr2 / Bispecific Antibody / OncologySelect advanced solid tumorsPhase 1OngoingGlobal
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- rezpegaldesleukinNKTR-358 / REZPEG
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- navacaprantNMRA-140 / Kappa opioid receptor (KOR) antagonist / Small molecule / NeuropsychiatryMajor Depressive Disorder
- NMRA-511NMRA-511 / Vasopressin 1a receptor (V1aR) antagonist / Small molecule / Neuropsychiatry / NeurodegenerativeAlzheimer's disease agitation
- NMRA-215NMRA-215 / NLRP3 inhibitor / Small molecule / Endocrinology / MetabolicObesity
- NMRA-861NMRA-861 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / NeuropsychiatrySchizophreniaNeuropsychiatric disorders
- NMRA-898NMRA-898 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / NeuropsychiatrySchizophreniaNeuropsychiatric disorders
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- triamcinolone acetonideZILRETTA / Small Molecule Formulation / Musculoskeletal PainShoulder Osteoarthritis (OA)Phase 3Active
- iovera°iovera° / Device / Neurology/PainSpasticityClinicalActive
- bupivacaine liposomeEXPAREL / Small Molecule Formulation / Pain ManagementAcute Post-Surgical Pain (Pediatric 0 to < 6)ClinicalActive
- enekinragene inzadenovecPCRX-201 / IL-1Ra / Gene Therapy / Musculoskeletal PainOsteoarthritis of the Knee (OAK)Phase 2Active
- PCRX-2002PCRX-2002 / Unknown / Pain ManagementPost-Surgical PainClinicalActive
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- VAX-31VAX-31 / Streptococcus pneumoniae (31 Serotypes) / Pneumococcal Conjugate Vaccine (PCV) / Infectious DiseasesInvasive 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 DiseasesInvasive Pneumococcal Disease (IPD) in InfantsPhase 2OngoingUnited States
- VAX-A1VAX-A1 / Group A Streptococcus / Conjugate Vaccine / Infectious DiseasesGroup A Strep InfectionsPreclinicalOngoing
- VAX-GIVAX-GI / Shigella / Vaccine / Infectious DiseasesShigellosis / Shigella InfectionsPreclinicalOngoing
- VAX-XLVAX-XL / Streptococcus pneumoniae / Pneumococcal Conjugate Vaccine (PCV) / Infectious DiseasesInvasive Pneumococcal Disease (IPD)PreclinicalOngoing
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- PAPZIMEOSzopapogene imadenovec-drba
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- PMN310PMN310 / Amyloid-Beta / Monoclonal Antibody / NeurologyAlzheimer's Disease
- PMN267PMN267 / TDP-43 / Monoclonal Antibody / NeurologyAmyotrophic Lateral Sclerosis (ALS)Frontotemporal Dementia (FTD)Limbic-predominant age-related TDP-43 encephalopathy (LATE)
- PMN442PMN442 / Alpha-Synuclein / Monoclonal Antibody / NeurologyParkinson's Disease (PD)Multiple System Atrophy (MSA)Dementia with Lewy Bodies (DLB)
- PMN311PMN311 / Amyloid-Beta / Vaccine / NeurologyAlzheimer's Disease
- PMN440PMN440 / Alpha-Synuclein / Vaccine / NeurologyParkinson's Disease
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- deupirfenidoneLYT-100 / Small Molecule / Respiratory / Fibrotic DiseasesIdiopathic Pulmonary Fibrosis (IPF)Phase 3ActiveGlobal
- LYT-200LYT-200 / Biologic / OncologyRelapsed/Refractory High-Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)Phase 1ActiveGlobalSolid TumorsPhase 1ActiveGlobal
- GlyphAlloSPT-300 / Allopregnanolone / Small Molecule / NeuropsychiatryAnxiety and Mood DisordersPhase 2ActiveGlobal
- GlyphAgoSPT-320 / Small Molecule / NeuropsychiatryNeurological / Neuropsychiatric IndicationsPhase 1ActiveGlobal
- xanomeline and trospium chlorideKarXT / Cobenfy / Muscarinic Receptors / Small Molecule / NeuropsychiatrySchizophreniaApprovedActiveUS
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- AMT-130AMT-130 / Huntingtin protein (mHTT) / Gene Therapy (AAV5) / Central Nervous SystemHuntington's DiseasePhase 1/2ActiveGlobal
- AMT-191AMT-191 / Alpha-galactosidase A (GLA) / Gene Therapy / Rare DiseasesFabry DiseasePhase 1/2aActiveGlobal
- AMT-260AMT-260 / Undisclosed / Gene Therapy / Central Nervous SystemRefractory Mesial Temporal Lobe EpilepsyPhase 1/2aActiveGlobal
- AMT-162AMT-162 / SOD1 / Gene Therapy / Central Nervous SystemAmyotrophic Lateral Sclerosis (ALS)Preclinical/ClinicalActiveGlobal
- etranacogene dezaparvovecAMT-061 / HEMGENIX / Factor IX / Gene Therapy (AAV5) / HematologyHemophilia BApprovedMarketedUSGlobal
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- BrepocitinibJAK1 / TYK2 / Small Molecule / Autoimmune & InflammatoryDermatomyositisPhase 3ActiveGlobalNon-infectious UveitisPhase 3ActiveGlobalCutaneous SarcoidosisPhase 2ActiveGlobal
- IMVT-1402IMVT-1402 / FcRn / Monoclonal Antibody / AutoimmuneRheumatoid Arthritis (difficult-to-treat)Phase 2ActiveGlobalGraves' DiseasePhase 2/3ActiveGlobal
- BatoclimabFcRn / Monoclonal Antibody / AutoimmuneThyroid Eye Disease (TED)Phase 3ActiveGlobalMyasthenia Gravis (MG)Phase 3ActiveGlobalChronic Inflammatory Demyelinating Polyneuropathy (CIDP)Phase 2bActiveGlobal
- MosliciguatRVT-304 / sGC / Small Molecule / PulmonaryPulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)Phase 2ActiveGlobal
- NamilumabGM-CSF / Monoclonal Antibody / Autoimmune & InflammatorySarcoidosisPhase 2ActiveGlobal
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- RopidoxuridineIPdR / Radiation Sensitizer / Small Molecule / OncologyGlioblastomaPhase 2ActiveUnited StatesSarcomasPhase 1/Phase 2ActiveUnited States
- SP-2-225SP-2-225 / HDAC6 / Small Molecule / OncologyBreast Cancer / Solid TumorsPreclinicalActiveUnited States
- SP-1-303SP-1-303 / Class I HDAC / Small Molecule / OncologyER+ Breast CancerPreclinicalActiveUnited States
- SP-1-161SP-1-161 / HDAC / Small Molecule / OncologySolid TumorsDiscoveryActiveUnited States
- PSMA-B ligandPSMA-B / PSMA / Radiopharmaceutical / OncologyMetastatic Prostate CancerPreclinicalActiveUnited States
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- GlyphAlloSPT-300 / Allopregnanolone prodrug
- GlyphAgoSPT-320 / Agomelatine prodrug
- Glyph2BLSDSPT-348 / Non-hallucinogenic neuroplastogen prodrug
- GlyphCeleCelecoxib prodrug
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- VeligrotugVRDN-001
- ElegrobartVRDN-003
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- MR-146MR-146 / AAV Gene Therapy / OphthalmologyNeurotrophic keratopathy (NK)
- Low Dose Estrogen Weekly PatchEstrogen / Progestin receptors / Transdermal Patch / Women's HealthContraception
- PitolisantHistamine H3 receptor / Small molecule / NeurologyObstructive Sleep Apnea Syndrome
- SelatogrelP2Y12 receptor / Small molecule / CardiovascularAcute Myocardial Infarction
- CenerimodS1P1 receptor / Small molecule / ImmunologySystemic lupus erythematosus (SLE)
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- WVE-006WVE-006 / AAT transcript (Pi*ZZ mutation) / RNA editing oligonucleotide (AIMer) / Genetic/Respiratory/Liver DiseasesAlpha-1 antitrypsin deficiency (AATD)
- WVE-007WVE-007 / INHBE mRNA / siRNA / Metabolic DiseasesObesity
- WVE-008WVE-008 / PNPLA3 I148M / RNA editing oligonucleotide / Liver DiseasesPNPLA3-related liver disease
- WVE-N531WVE-N531 / DMD Exon 53 / Exon skipping oligonucleotide / Neuromuscular DiseasesDuchenne muscular dystrophy (DMD)
- WVE-003WVE-003 / Mutant huntingtin (mHTT) mRNA / Allele-selective silencing oligonucleotide / Neurological DiseasesHuntington's disease (HD)
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- ABCL635ABCL635 / NK3R / antibody medicine / Endocrinology / Women's HealthVasomotor Symptoms (VMS) associated with menopausePhase 2active
- ABCL575ABCL575 / OX40L / monoclonal antibody / Immunology & InflammationAtopic Dermatitis (AD)Phase 1active
- ABCL688ABCL688 / Undisclosed GPCR / Ion Channel / antibody medicine / AutoimmunityUndisclosed autoimmunity indicationIND-Enablingactive
- ABCL386ABCL386 / Undisclosed / antibody medicine / OncologyUndisclosed oncology indicationIND-Enablingactive
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- prademagene zamikeracelpz-cel / ZEVASKYN / COL7A1 / autologous cell-based gene therapy / Dermatology / rare diseaserecessive dystrophic epidermolysis bullosa (RDEB)Approvedapproved
- PSMA SIR-TABO-701 / PSMA / engineered autologous T-cell therapy / OncologyAdvanced prostate cancerPreclinicalpreclinical
- UX111UX111 / SGSH / in vivo AAV9 gene therapy / Rare disease / CNSSanfilippo Syndrome Type A (MPS IIIA)Phase 3clinical
- TSHA-102TSHA-102 / MECP2 / intrathecal AAV9 gene transfer therapy / Rare disease / CNSRett SyndromePhase 1/2clinical
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- MitapivatPYRUKYND®; AQVESME™ / PK / PK activator / Rare diseases; hematologyAdult PK deficiencyApprovedAdult thalassemiaApprovedU.S.KSAUAEAdult sickle cell diseaseRegulatory submissionU.S.
- TebapivatPK / PK activator / Rare diseases; hematologyLower-risk myelodysplastic syndromesClinical developmentSickle cell diseaseClinical development
- AG-181AG-181 / PAH / PAH stabilizer / Rare diseases; metabolic disordersPhenylketonuriaClinical development
- AG-236AG-236 / TMPRSS6 / siRNA / Rare diseases; hematologyPolycythemia veraClinical development
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- PraliciguatsGC / soluble guanylate cyclase stimulator / Kidney diseaseFocal segmental glomerulosclerosisPhase 2first patient enrolled Dec 2025
- Ebribafusp alfaAKB-097 / C3d/factor H / anti-C3d-factor H fusion protein; complement inhibitor / Rare kidney diseasesIgA 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 careCardiac surgery-associated acute kidney injuryPhase 1first participants dosed Apr 2026Acute respiratory distress syndromePreclinical
- AKB-10108AKB-10108 / HIF-PH / HIF-PH inhibitor / Other disease areasRetinopathy of prematurityPreclinical
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- EnvudeucitinibESK-001 / TYK2 / oral small molecule TYK2 inhibitor / ImmunologyPlaque 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 / NeuroinflammationMultiple sclerosisPhase 1 completed; Phase 2 plannedPhase 2 initiation planned H1 2026
- LonigutamabIGF-1R / subcutaneous humanized IgG1 monoclonal antibody / Endocrinology/ImmunologyThyroid eye diseaseDevelopment program evaluation ongoing
- IRF5/Additional TargetsIRF5, additional targetsUndisclosed
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- AvexitideGLP-1 receptor / GLP-1 receptor antagonist / Endocrine/metabolic diseasesPost-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 diseasesWolfram syndromePhase 2HELIOS positive Week 48 data; pivotal Phase 3 planned pending FDA alignmentUSEU
- AMX0114AMX0114 / Calpain-2 (CAPN2) / Antisense oligonucleotide (ASO) / Neurodegenerative diseasesAmyotrophic 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 diseasesPBH 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 inflammationAtopic 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 inflammationAtopic dermatitisPhase 1in-progress; Phase 1b PoC fully enrolled
- APG273APG273 / IL-13 + TSLP / combination of zumilokibart (APG777) + APG333 monoclonal antibodies / respiratory immunology and inflammationAsthma / COPDPreclinicalin-progress
- APG808APG808 / IL-4Rα / antibody / respiratory immunology and inflammationAsthmaPhase 1bpositive interim results announced May 2025
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- BPL-003BPL-003 / Intranasal mebufotenin benzoate / Mental healthTreatment-resistant depressionPhase 2In development
- VLS-01VLS-01 / 5-HT1/2/6/7 receptor agonist / Oral transmucosal DMT buccal film / Mental healthTreatment-resistant depressionPhase 2In development
- EMP-01EMP-01 / Oral R-MDMA / Mental healthSocial anxiety disorderPhase 2Positive topline results announced
- Novel 5-HT2A Receptor Agonists5-HT2A receptor / Small-molecule neuroplastogens / Mental healthOpioid use disorderDiscoveryIn developmentTreatment-resistant depressionDiscoveryIn development
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- DeramiocelCAP-1002 / allogeneic CDCs / Neuromuscular diseaseDuchenne muscular dystrophy (DMD)Phase 3in progressBecker muscular dystrophy (BMD)Preclinicalin progress
- StealthX™STX-S / Exosome-based vaccine / Infectious diseaseSARS-CoV-2Phase 1in progress
- StealthX™Therapeutic ExosomesEvaluatingPreclinicalin progress
- CDC-ExosomesExosome Platform / Neuromuscular diseaseDuchenne muscular dystrophyPreclinicalin progress
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- CLN-978CLN-978 / CD19xCD3 / bispecific T cell engager / ImmunologyOUTRACE: 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 / ImmunologyAutoimmune 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) / OncologyREZILIENT1: 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 / OncologyAcute 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 / OncologyRelapsed platinum-sensitive ovarian cancer; maintenance therapyAdaptive platform trialongoing
- COM701 + COM902 + pembrolizumabPVRIG; TIGIT; PD-1 / Antibody combination / OncologyPlatinum-resistant ovarian cancerProof of concept
- rilvegostomigPD-1/TIGIT / Fc-reduced anti-PD-1/TIGIT bispecific antibody / OncologyNSCLCPhase 3; Phase 1/2ongoingGastrointestinal cancerPhase 3; Phase 1/2ongoingEndometrial cancerPhase 3ongoing
- GS-0321GS-0321 / IL18BP / Anti-IL18BP antibody / OncologySolid tumorsPhase 1ongoing
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- CNM-Au8®CNM-Au8 / mitochondrial function / NAD pathway; neuroprotection and remyelination / oral gold nanocrystal suspension / NeurologyAmyotrophic 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 diseaseAntiviral and antimicrobial usesDemonstrated antiviral and antimicrobial properties
- CNM-AgZn17CNM-AgZn17 / topical gel polymer suspension of silver and zinc ions / Infectious disease / wound healingInfectious diseasesIn developmentWound healingIn development
- CNM-PtAu7CNM-PtAu7 / gold-platinum nanotherapeutic / OncologyCancerIn vitroInitial research
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- tovecimigCTX-009 / DLL4 x VEGF-A / bispecific antibody / OncologyBiliary 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 / OncologyNCAM (CD56) basket studyTrial initiation expected Q1 2026Post-checkpoint basket studyCompleted
- CTX-8371CTX-8371 / PD-1 x PD-L1 / bispecific antibody / OncologySolid tumorsPhase 1Expansion cohorts initiated; Phase 1 data expected H1 2026
- CTX-10726CTX-10726 / PD-1 x VEGF-A / bispecific antibody / OncologySolid tumorsIND-enabling preclinicalPhase 1 initiation expected Q1 2026
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- PIPE-791LPA1R / small molecule LPA1R antagonist / inflammation; immunology; neuroscienceIdiopathic pulmonary fibrosisPhase 2activeProgressive MSPhase 1deferredChronic painPhase 1bactive
- CTX-343LPA1R / peripherally restricted small molecule LPA1R antagonist / peripheral indicationsPeripheralPreclinicaldeferred
- PIPE-307JNJ-5120 / M1R / small molecule M1R antagonist / neuroscienceRelapsing-remitting MSPhase 2licensedMajor depressive disorderPhase 2licensed
- CalpainCalpain / calpain inhibitorUndisclosedPreclinicalactive
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- AficamtenMYQORZO / cardiac myosin inhibitor / small molecule / CardiovascularoHCMApproved & CommercializedApproved in U.S., China, & EUU.S.ChinaEUoHCM (vs. metoprolol)Phase 3MAPLE-HCMPediatric oHCMPhase 2CEDAR-HCM
- Ulacamtencardiac myosin inhibitor / small molecule / CardiovascularHFpEFPhase 2AMBER-HFpEF
- Omecamtiv Mecarbilcardiac myosin activator / small molecule / CardiovascularHFrEFPhase 3COMET-HF
- CK-089CK-089 / fast skeletal muscle troponin activator / small molecule / NeuromuscularMuscular Dystrophy, OtherPhase 1
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- DT-216P2DT-216P2 / FXN GAA repeat expansion / frataxin expression / GeneTAC small molecule / Genetic disease / neurologyFriedreich 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 diseaseFuchs 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 diseaseMyotonic 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 diseaseHuntington's diseasePreclinicalProgram disclosed; candidate not named
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- Halneuron® NaV1.7 InjectionHAL / Halneuron® / NaV1.7 / subcutaneous small-molecule/peptide analgesic injection / Pain / neuropathyChemotherapy-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 painChemotherapy-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 illnessFibromyalgiaPhase 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 illnessLong 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 dystrophyBecker muscular dystrophyPivotal / Phase 3Duchenne muscular dystrophyPhase 2
- EDG-7500EDG-7500 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / CardiovascularHypertrophic cardiomyopathyPhase 2
- EDG-15400EDG-15400 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / CardiovascularHFpEFPhase 1
- EDG-003EDG-003 / Undisclosed target / CardiometabolicCardiometabolicPreclinical
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- 4D-1504D-150 / VEGF family members / Intravitreal AAV genetic medicine / RetinaWet 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 / PulmonologyCystic 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 / RetinaGeographic atrophySeeking partnerships to advance
- 4D-7254D-725 / Aerosol-delivered AAV genetic medicine / PulmonologyAlpha-1 antitrypsin lung diseasePreclinicalSeeking partnerships to advance
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- MRT-6160MRT-6160 / VAV1 / Oral molecular glue degrader / Immunology & InflammationImmune-mediated diseasesClinical
- MRT-8102MRT-8102 / NEK7 / Oral molecular glue degrader / Immunology & InflammationIL-1β/NLRP3-driven inflammatory diseasesPhase 1
- MRT-2359MRT-2359 / GSPT1 / Oral molecular glue degrader / OncologyCastration-resistant prostate cancerPhase 1/2Ongoing
- Cyclin E1 degrader programCCNE1 / CDK2 / Molecular glue degrader / OncologyCCNE1-amplified tumorsPreclinicalLead optimizationER+ breast cancerPreclinicalLead optimization
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- HCW9302HCW9302 / IL-2Rα / IL-2 fusion protein / AutoimmuneAlopecia areataPhase 1OngoingUS
- HCW11-006HCW11-006 / Multiple cytokines (IL-15/IL-15Rα, IL-7, IL-21, IL-2) / Multi-functional fusion immunotherapeutic / OncologyCancerPhase 1Planned by licenseeChina
- HCW11-018bHCW11-018b / Tissue Factor/CD3 / Tetra-valent bispecific T-cell engager (Big BiTE) / OncologySolid tumorsPreclinicalIND-enablingGlobal
- HCW11-040HCW11-040 / PD-1 (pembrolizumab-based) + TGF-β / Tetra-valent immune checkpoint inhibitor fusion / OncologySolid tumorsPreclinicalIND-enablingGlobal
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- nogapendekin alfa inbakiceptN-803 / ANKTIVA® / IL-15 receptor / Cytokine fusion protein / Interleukin-15 superagonist / Oncology, Infectious DiseasesBCG 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 / OncologyAdvanced Solid TumorsPhase 2ActiveUS
- AldoxAlbumin-bound doxorubicin complex / OncologyAdvanced Solid TumorsPhase 2ActiveUS
- Recombinant BCGVaccine / Immunotherapy / OncologyBladder CancerExpanded AccessActiveUS
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- anzu-cel (IMA203)IMA203 / anzutresgene autoleucel / PRAME / Autologous TCR-T cell therapy / Solid Tumors2L+ cutaneous melanomaPhase 3ongoingUSEU5Uveal melanomaPhase 1bongoingUSEU5PRAME+ solid tumorsPhase 1ongoingUSEU5
- IMA203CD8 (GEN2)IMA203CD8 / PRAME / Autologous TCR-T cell therapy (next-gen with CD8) / Solid TumorsPRAME+ advanced solid tumorsPhase 1dose escalation ongoingUSEU5Ovarian and endometrial cancersPhase 1ongoingUSEU5
- IMA402IMA402 / PRAME / Half-life extended TCR bispecific (TCER) / Solid TumorsRecurrent/refractory solid tumors (melanoma, gynecologic, sqNSCLC)Phase 1/2Phase 1b expansion ongoingUSEU
- IMA401IMA401 / MAGEA4/8 / TCR bispecific (TCER) / Solid TumorsRecurrent/refractory solid tumors (HNSCC, sqNSCLC, melanoma)Phase 1dose escalation completedUSEU
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- INB-100INB-100 / DeltEx Allo allogeneic γδ T cell therapy / OncologyAcute leukemia post-HSCTPhase 1Expansion cohort enrollingUS
- INB-200INB-200 / DeltEx DRI autologous genetically modified γδ T cell therapy / OncologyNewly diagnosed glioblastomaPhase 1Treatment completed; long-term follow-upUS
- INB-400INB-400 / DeltEx DRI autologous and allogeneic γδ T cell therapy / OncologyNewly diagnosed glioblastomaPhase 2Enrollment suspended; treatment completed; data collection ongoingUS
- INB-619INB-619 / CD19 / γδ T cell engager (TCE) / Oncology and autoimmune diseasesCD19+ 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 ImmunologyCritical 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 / OncologySolid tumors (NSCLC, CRC liver metastases)PreclinicalIND-enabling studies underway
- MiNK-413MiNK-413 / BCMA / IL-15 armored BCMA-CAR-iNKT cell therapy / OncologyMultiple myelomaPreclinical
- PRAME TCR-iNKTPRAME / TCR-engineered iNKT cell therapy / OncologyPediatric cancers/solid tumorsPreclinicalIND-enabling
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- bitopertinGlyT1 / Small Molecule / HematologyErythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP)Phase 3OngoingGlobalDiamond-Blackfan AnemiaPhase 2OngoingUS
- DISC-0974DISC-0974 / Hemojuvelin (HJV) / Monoclonal Antibody / HematologyAnemia 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 / HematologyPolycythemia Vera (PV)Phase 2OngoingGlobalSickle Cell Disease (SCD)Phase 1bOngoingGlobal
- DISC-0998DISC-0998 / Hemojuvelin (HJV) / Monoclonal Antibody / HematologyInflammatory-disease-associated anemiaPreclinicalOngoingGlobal
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- SelinexorKPT-330 / XPOVIO / XPO1 (Exportin 1) / Small Molecule / OncologyMultiple MyelomaMarketedActiveUSGlobalEndometrial CancerPhase 3ActiveUSGlobalMyelofibrosisPhase 3ActiveUSGlobal
- EltanexorKPT-8602 / XPO1 (Exportin 1) / Small Molecule / OncologyMyelodysplastic NeoplasmsPhase 2PausedUSColorectal CancerPhase 2PausedUSProstate CancerPhase 2PausedUS
- KPT-9274KPT-9274 / PAK4 / NAMPT / Small Molecule / OncologySolid Tumors & LymphomaPhase 1PausedUS
- VerdinexorKPT-335 / XPO1 (Exportin 1) / Small Molecule / Veterinary Oncology / Infectious DiseasesCanine LymphomaMarketedActiveUS
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- IFx-2.0IFx-Hu2.0 / Innate Immune System / Personalized Cancer Vaccine / Innate Immune Agonist / OncologyAdvanced 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 / OncologyAdvanced 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) / OncologySolid TumorsPreclinicalOngoingUnited States
- REM-001Unknown / Photodynamic Therapy / OncologyCutaneous Metastatic Breast Cancer (CMBC)Phase 3OngoingUnited States
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- PAS-004PAS-004 / MEK 1/2 / Macrocyclic Small Molecule / Oncology / Rare DiseasesNeurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN)MAPK pathway-driven advanced solid tumors
- PAS-003PAS-003 / α5β1 Integrin / Monoclonal Antibody / NeurologyAmyotrophic Lateral Sclerosis (ALS)
- PAS-001PAS-001 / C4A / Small Molecule / PsychiatrySchizophrenia
- PAS-002PAS-002 / NeurologyMultiple Sclerosis (MS)
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- ZiftomenibKO-539 / KOMZIFTI / Menin-KMT2A/MLL interaction / Small molecule / Hematologic MalignanciesR/R NPM1-mutated AMLNewly Diagnosed AMLFLT3-mutated AML
- DarlifarnibKO-2806 / Farnesyl transferase / Small molecule / OncologyClear cell renal cell carcinoma (ccRCC)Solid tumors
- TipifarnibFarnesyl transferase / Small molecule / OncologyHRAS-mutant Head and Neck Squamous Cell Carcinoma (HNSCC)PIK3CA-dependent HNSCC
- KO-7246KO-7246 / Menin / Small molecule / CardiometabolicDiabetesCardiometabolic disease
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- SAT-3247SAT-3247 / AAK1 / Small molecule / Degenerative muscle diseasesDuchenne Muscular Dystrophy (DMD)Facioscapulohumeral Muscular Dystrophy (FSHD)
- SAT-3153SAT-3153 / Notch polarity kinase / Small molecule / Degenerative muscle diseasesDuchenne Muscular Dystrophy (DMD)
- BertilimumabiCo-008 / Eotaxin-1 (CCL11) / Monoclonal antibody / ImmunologyBullous Pemphigoid
- iCo-019iCo-019 / Fungal cell membrane / Small molecule (Oral Amphotericin B) / Infectious DiseasesFungal infections
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- GivastomigCLDN18.2 x 4-1BB / Bispecific Antibody / OncologyGastric CancerGastrointestinal Malignancies
- RagistomigPD-L1 x 4-1BB / Bispecific Antibody / OncologySolid Tumors
- UliledlimabCD73 / Monoclonal Antibody / OncologyCancer
- VIS-101VEGF-A x ANG-2 / Bispecific Antibody / OphthalmologyWet Age-Related Macular Degeneration (AMD)Diabetic Macular Edema (DME)
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- NEO100NEO-100 / Unknown / Small Molecule / Oncology, NeurologyMalignant GliomasGrade 4 AstrocytomaParkinson's Disease
- NEO212NEO-212 / DNA / Small Molecule Conjugate / OncologyMalignant Gliomas
- NEO214NEO-214 / DR5, PDE4 / Small Molecule Conjugate / OncologyGlioblastoma (GBM)Multiple Myeloma
- NEO400NEO-400 / Unknown / Small Molecule Conjugate / Dermatology, OncologyUV-Induced Skin DamageTopical Oncology
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- TaletrectinibIBTROZI / ROS1 / Small Molecule / OncologyAdvanced ROS1+ NSCLC
- SafusidenibmIDH1 / Small Molecule / OncologyDiffuse IDH1-mutant gliomaAstrocytomaOligodendroglioma
- NUV-1511NUV-1511 / Undisclosed / Drug-Drug Conjugate (DDC) / OncologyAdvanced solid tumors
- NUV-868NUV-868 / BET (BD2-selective) / Small Molecule / OncologyAdvanced solid tumors
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- NarsoplimabOMS721 / Yartemlea / MASP-2 / Monoclonal antibody / Hematology / Complement-Mediated DiseasesHematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)
- ZaltenibartOMS906 / MASP-3 / Monoclonal antibody / Hematology / NephrologyParoxysmal nocturnal hemoglobinuria (PNH)C3 glomerulopathy
- OMS1029OMS1029 / MASP-2 / Biologic / Complement-Mediated Diseases
- OMS527OMS527 / PDE7 / Small molecule / AddictionCocaine use disorder
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- leniolisibJoenja
- napazimoneKL1333
- conestat alfaRUCONEST
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- UlixacaltamidePRAX-944 / T-type calcium channel / Small molecule / NeurologyEssential Tremor
- RelutriginePRAX-562 / Persistent sodium current (NaV) / Small molecule / NeurologyDevelopmental and Epileptic Encephalopathies (DEEs)SCN2A-DEESCN8A-DEE
- VormatriginePRAX-628 / Sodium channels (NaV) / Small molecule / NeurologyFocal Onset EpilepsyEpilepsy
- ElsunersenSCN2A mRNA / Antisense oligonucleotide (ASO) / NeurologySCN2A Developmental and Epileptic Encephalopathy
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- ABBV-RGX-314surabgene lomparvovec / sura-vec
- RGX-121clemidsogene lanparvovec / NAVSUNLI
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- ZovegalisibRLY-2608 / Mutant PI3Kα / Small molecule / Oncology & Genetic DiseaseHR+/HER2- metastatic breast cancer (PI3Kα-mutated)Phase 3ActiveGlobalPIK3CA-driven vascular malformations/anomaliesPhase 1ActiveGlobal
- LirafugratinibRLY-4008 / FGFR2 / Small molecule / OncologyFGFR2-altered solid tumorsClinicalOut-licensedGlobal
- RLY-8161RLY-8161 / NRAS / Small molecule / OncologyNRAS-driven solid tumorsPreclinicalActiveGlobal
- Fabry disease programUndisclosed / Non-inhibitory chaperone / Genetic DiseaseFabry diseasePreclinicalActiveGlobal
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- IMCIVREEsetmelanotide
- bivamelagonLB54640
- Preclinical small moleculesCongenital hyperinsulinism (CHI)
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- SCY-770PXL-770 / AMP-activated protein kinase (AMPK) / Small Molecule / Rare Diseases / NephrologyAutosomal Dominant Polycystic Kidney Disease (ADPKD)Phase 2ActiveGlobal
- SCY-247SCY-247 / 1,3-beta-glucan synthase / Small Molecule / Infectious DiseasesTreatment and Prevention of Invasive Fungal InfectionsPhase 1ActiveUnited States
- Fungerp analogs1,3-beta-glucan synthase / Small Molecule / Infectious DiseasesTreatment of resistant fungiPreclinicalActiveGlobal
- IbrexafungerpSCY-078 / BREXAFEMME / 1,3-beta-glucan synthase / Small Molecule / Infectious DiseasesVulvovaginal candidiasis (VVC) and Recurrent VVCApprovedActiveUnited StatesInvasive Candidiasis (IC)Phase 3ActiveGlobal
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- SEP-479SEP-479 / PTH1R / Small Molecule / EndocrinologyHypoparathyroidismPhase 1Active
- SEP-631SEP-631 / MRGPRX2 / Small Molecule / Immunology and InflammationChronic spontaneous urticaria (CSU)Phase 1ActiveOther mast cell diseasesPhase 1Active
- TSHR ProgramTSHR / Small Molecule / EndocrinologyGraves' diseasePreclinicalActiveThyroid eye diseasePreclinicalActive
- Novo Nordisk Partnered ProgramsGLP-1R, GIPR, GCGR, Undisclosed / Small Molecule / Metabolic DiseasesMetabolic diseasesDiscoveryActive
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- SPC-15SPC-15 / 5-HT4 receptor agonist / Small Molecule / PsychiatryPTSD and stress-induced anxiety disordersPreclinicalActiveGlobal
- SP-26SP-26 / NMDA receptor antagonist / Implant / NeurologyFibromyalgia and chronic painPreclinicalActiveGlobal
- SPC-14SPC-14 / Unknown / Small Molecule / NeurologyAlzheimer's diseasePreclinicalActiveGlobal
- SPU-16SPU-16 / Unknown / Peptide / NeurologyMultiple sclerosisPreclinicalActiveGlobal
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- patidegibSGT-610 / Hedgehog signaling pathway (SMO) / Small molecule / DermatologyGorlin syndrome (prevention of new BCC lesions)High-frequency basal cell carcinoma (BCC)
- erlotinibSGT-210 / EGFR / Small molecule / DermatologyUndisclosed indications
- tretinoin and benzoyl peroxide cream, 0.1%/3%TWYNEO / Retinoid receptors / Bacterial proteins / Small molecule / DermatologyAcne vulgaris
- benzoyl peroxide cream, 5%EPSOLAY / Bacterial proteins / Small molecule / DermatologyInflammatory lesions of rosacea
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- TSC-100TSC-100 / HA-1 / TCR-T Cell Therapy / Hematologic MalignanciesAcute 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 MalignanciesAcute 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 / OncologySolid TumorsPhase 1ActiveUnited States
- Novel TargetsUnidentified Autoantigens / Targeted Therapeutics / Autoimmune DisordersCrohn's DiseaseDiscoveryActiveUnited States
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- VXA-CoV2-3.3VXA-CoV2-3.3 / SARS-CoV-2 / Oral recombinant adenovirus vaccine / Infectious DiseasesCOVID-19
- Norovirus Oral Vaccine CandidateNorovirus (GI.1 and GII.4) / Oral recombinant adenovirus vaccine / Infectious DiseasesNorovirus InfectionAcute Gastroenteritis
- Seasonal Influenza Oral Vaccine CandidateVXA-A1 / Influenza Virus / Oral recombinant adenovirus vaccine / Infectious DiseasesSeasonal Influenza
- HPV Therapeutic Vaccine CandidateHuman Papillomavirus (HPV) / Oral recombinant therapeutic vaccine / OncologyHPV-associated Cancers / Immuno-oncology
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- AzetukalnerXEN1101 / Kv7.2/Kv7.3 potassium channel opener / Small Molecule / Neurology and PsychiatryFocal Onset Seizures (FOS)Phase 3OngoingGlobalPrimary Generalized Tonic-Clonic Seizures (PGTCS)Phase 3OngoingGlobalMajor Depressive Disorder (MDD)Phase 3OngoingGlobal
- XEN1120XEN1120 / Kv7 potassium channel opener / Small Molecule / PainPainPhase 1OngoingUnited States
- XEN1701XEN1701 / NaV1.7 sodium channel modulator / Small Molecule / PainPainPhase 1OngoingUnited States
- NBI-921355NBI-921355 / NaV1.2 / NaV1.6 sodium channel inhibitor / Small Molecule / NeurologyEpilepsyPhase 1OngoingGlobal
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- SabirnetugACU193 / toxic soluble amyloid beta oligomers (AβOs) / humanized monoclonal antibody; intravenous formulation / NeurologyEarly Alzheimer's diseasePhase 2ongoing ALTITUDE-ADGlobal
- SabirnetugACU193 / toxic soluble amyloid beta oligomers (AβOs) / humanized monoclonal antibody; subcutaneous formulation / NeurologyEarly Alzheimer's diseasePhase 1clinical development
- Enhanced Brain Delivery (EBD™)TfR brain transporter + AβO mAb / blood-brain-barrier-penetrating antibody therapy / NeurologyEarly Alzheimer's diseasePreclinicalpreclinicalPre-clinical Alzheimer's diseasePreclinicalpreclinical
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- Sodium oxybate extended-release oral suspensiononce-nightly extended-release oral suspension / Sleep disordersIdiopathic hypersomniaPhase 3Evaluating in REVITALYZ
- Olanzapine and samidorphanLYBALVI / oral small molecule combination / PsychiatrySchizophrenia and bipolar I disorder (pediatric)Phase 3Evaluating in ENLIGHTEN-Youth
- AlixorextonALKS 2680 / orexin 2 receptor / oral small molecule agonist / Sleep disordersNarcolepsy type 1Phase 3Brilliance Studies programNarcolepsy type 2Phase 3Brilliance Studies programIdiopathic hypersomniaPhase 2Vibrance-3
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- APR-1051APR-1051 / WEE1 kinase / Oral small-molecule WEE1 inhibitor / OncologyCCNE1, 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 / OncologyATRi 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 / OncologySolid 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/immunologyType 1 allergic reactions, including anaphylaxisNDA resubmission plannedclinical/regulatoryUS
- AQST-108AQST-108 / epinephrine prodrug / epinephrine topical gel / DermatologyMultiple potential indicationsPhase 1clinical
- LibervantLibervant / GABA-A receptor positive allosteric modulator / diazepam buccal film / NeurologyAcute repetitive seizures / cluster seizuresRegulatory/approvedpipeline-listedUS
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- Topical Roflumilast CreamARQ-151 / ZORYVE / PDE4 / small molecule topical cream / DermatologyPlaque 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 / DermatologyVitiligo 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 / DermatologyAtopic 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; oncologyRefractory 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 / OncologyHematologic malignanciesPreclinicalcollaborator-funded
- CAR-NKAB-201 / HER2 / Allogeneic anti-HER2 CAR-NK cell therapy expressing soluble IL-15 / OncologySolid tumorsPreclinicalcollaborator-funded; US orphan drug designationUS
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- EfzofitimodNRP2 modulator / tRNA synthetase-derived biologic immunomodulator / Interstitial lung diseasePulmonary sarcoidosisPhase 3SSc-ILDPhase 2Other ILD (CTD-ILD; CHP)Phase 1
- ATYR0101ATYR0101 / LTBP1 modulator / FibrosisFibrosisPreclinical
- ATYR0750ATYR0750 / FGFR4 modulator / Liver disordersLiver disordersPreclinical
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- ATR-12ATR-12 / LEKTI replacement / SPINK5 / Topical live biotherapeutic; engineered S. epidermidis expressing rhLEKTI-D6 / DermatologyNetherton syndromePhase 1bclinicalUS
- ATR-04ATR-04 / IL-36g reduction; S. aureus inhibition / Topical live biotherapeutic; auxotrophic engineered S. epidermidis strain SE484 / Dermatology / oncology supportive careEGFR inhibitor-associated rashPhase 1/2clinicalUS
- ATR-01ATR-01 / Filaggrin / FLG / Topical protein candidate; FLG5-6 with cell-penetrating peptide / DermatologyIchthyosis vulgarisPreclinicalpreclinical
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- BGE - 102BGE-102 / NLRP3 / CNS-penetrant NLRP3 inhibitor / Metabolic diseasesCV riskPhase 1Diabetic macular edemaPhase 1
- APJ OralAPJ / APJ agonist / Metabolic diseasesObesityLead optimization
- APJ ParenteralAPJ / APJ agonist / Metabolic diseasesObesityLead optimization
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- VTP-1000VTP-1000 / gluten antigens; mTOR inhibitor co-delivery / antigen-specific immunotherapy; SNAP Tolerance Platform / AutoimmunityCeliac 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 diseasesChronic 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 / CancerProstate cancerPhase 1Pursuing partnership opportunities for advancementWorldwide rights
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- LOQTORZI (toripalimab-tpzi)LOQTORZI / PD-1 / Anti-PD-1 monoclonal antibody / Oncology1L Nasopharyngeal Carcinoma (combo with chemotherapy)ApprovedApprovedUnited States2L+ Nasopharyngeal Carcinoma (monotherapy)ApprovedApprovedUnited States
- TagmokitugCHS-114 / CCR8 / Anti-CCR8 monoclonal antibody / Oncology4L+ 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 / Oncology1L Hepatocellular Carcinoma (combo with toripalimab + bevacizumab)Phase 2
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- CTx-1301CTx-1301 / PTR multi-core modified-release dexmethylphenidate tablet / CNSADHDNDA submittedSubmitted July 31, 2025US
- CTx-1302CTx-1302 / PTR multi-core modified-release dextroamphetamine tablet / CNSADHDPreclinical/formulationInitial formulation study plannedUS
- CTx-2103CTx-2103 / 5-HT1A partial agonist / PTR multi-core modified-release buspirone tablet / CNSAnxietyPre-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 / OncologyRelapsed/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 / OncologyRelapsed/refractory triple-negative breast cancerPhase 1bongoing
- CLR 121225CLR 225 / tumor cells via phospholipid ether platform / PLE radioconjugate; actinium-225 alpha emitter / OncologyPancreatic cancerPhase 1planned
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- Budoprutug IVCD19 / monoclonal antibody / immune-mediated diseasesPrimary Membranous NephropathyPhase 2Immune ThrombocytopeniaPhase 1Systemic Lupus ErythematosusPhase 1
- Budoprutug SCCD19 / monoclonal antibody / immune-mediated diseasesAutoimmune DiseasePhase 1healthy volunteer trial
- CLYM116CLYM116 / APRIL / monoclonal antibody / immune-mediated diseasesIgA NephropathyPhase 1healthy volunteer trial
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- cleminorextonORX750 / OX2R / oral orexin receptor 2 agonist / Sleep-wake disordersNarcolepsy Type 1 (NT1)Phase 2aactiveNarcolepsy Type 2 (NT2)Phase 2aactiveIdiopathic Hypersomnia (IH)Phase 2aactive
- ORX142ORX142 / OX2R / oral orexin receptor 2 agonist / Neurology/neurodegenerative disordersNeurological & neurodegenerative disordersPhase 1active
- ORX489ORX489 / OX2R / oral orexin receptor 2 agonist / NeuropsychiatryNeuropsychiatric disordersPreclinicalactive
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- CTIM-76CTIM-76 / CLDN6 x CD3 / TCE bispecific antibody / Oncologyrecurrent ovarian cancer and other advanced solid tumorsPhase 1ongoingplatinum-resistant ovarian cancerPhase 1FDA Fast TrackUS
- CT-95CT-95 / MSLN x CD3 / TCE bispecific antibody / Oncologyadvanced solid tumorsPhase 1ongoing
- CT-202CT-202 / Nectin-4 x CD3 / TCE bispecific antibody / OncologyNectin-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; oncologyRelapsed/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 / OncologyCancer indicationsClinical-stageGreater China
- MupadolimabCPI-006 / CD73 / Anti-CD73 monoclonal antibody / OncologyNon-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 / Oncology3L+ 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 / OncologyAdvanced/metastatic melanomaPhase 1active
- CX-908CX-908 / P-Cadherin x CD3 / Probody T-cell engager / OncologySolid tumorsPreclinicalactive
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- HLP0035-HT2A and other serotonin receptors / Deuterated novel serotonergic agonist; oral / Mental healthAdjunctive treatment of Major Depressive Disorder (MDD)Phase 3Dosing underway; FDA Breakthrough Therapy Designation
- HLP004Serotonin receptors / Deuterated novel serotonergic agonist; intramuscular / Mental healthGeneralized Anxiety Disorder (GAD)Phase 2Enrollment complete
- HLP005CYB005 / Phenethylamines and tryptamines / CNS disordersCNS DisordersPreclinical
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- TovorafenibDAY101 / OJEMDA / RAF / MAPK pathway / Oral small-molecule type II RAF inhibitor / OncologyBRAF-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) / OncologyAdenoid 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) / OncologyAdult 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 diseasesVitiligoPhase 2Health Canada CTA approved; IND in processCanadaUnited States
- EB01 (daniluromer)EB01 / sPLA2 / Topical sPLA2 inhibitor / Dermatological diseasesAllergic contact dermatitisPhase 2b completedTopline results available
- ParidiprubartEB05 / TLR4 / Monoclonal antibody / Respiratory diseasesAcute 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 / OncologyRAS-mutant solid tumorsPhase 1AURORAS-1; in clinicWorldwide
- ERAS-4001ERAS-4001 / KRAS / Small molecule / OncologyKRAS-mutant solid tumorsPhase 1BOREALIS-1; in clinicWorldwide
- ERAS-12ERAS-12 / EGFR D2/D3 / Large molecule; bispecific antibody / OncologyEGFR 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/oncologymutCALR-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/oncologymutCALR-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/oncologymutCALR-driven myeloproliferative neoplasmsPreclinical / IND-enablingIND or CTA submission planned 2027USex-US
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- quaratusugene ozeplasmid (REQORSA)REQORSA / TUSC2 / non-viral gene therapy / OncologyEGFR-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 / DiabetesType 1 DiabetesPreclinicalType 2 DiabetesPreclinical
- NPRL2 gene therapyNPRL2 / non-viral gene therapy / OncologyKRAS/STK11 mutant anti-PD1-resistant NSCLCPreclinical
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- HLP0035-HT2A (and other serotonin receptors) / Deuterated NSA (oral) / Mental HealthAdjunctive treatment of major depressive disorder (MDD)Phase 3Dosing underway (PARADIGM program); FDA Breakthrough Therapy DesignationUnited StatesEuropeAustralia
- HLP004Serotonin receptors / Deuterated NSA (intramuscular) / Mental HealthGeneralized anxiety disorder (GAD)Phase 2Positive topline results reportedUnited States
- HLP005NSA (phenethylamines and tryptamines) / Mental HealthCNS disordersPreclinical
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- IFx-2.0plasmid DNA (innate immune agonist) / OncologyAdvanced 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 / OncologyNPM1-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) / OncologyAcute myeloid leukemia (AML)PreclinicalLead selection
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- Vidofludimus calciumIMU-838 / DHODH inhibitor and Nurr1 activator / Oral small molecule / Multiple sclerosisRelapsing 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 diseasesCeliac diseasePhase 1bCompleted (positive proof-of-concept)Global
- IMU-381IMU-381 / Nurr1 / Oral small molecule / Autoimmune diseasesNeurologic, gastrointestinal and other autoimmune diseasesPreclinicalPreclinical
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- tarcocimab tedromerZenkuda / VEGF / Antibody Biopolymer Conjugate (ABC) / OphthalmologyWet Age-Related Macular Degeneration (wet AMD)Diabetic RetinopathyRetinal Vein Occlusion (RVO)
- KSI-501KSI-501 / IL-6 / VEGF / Bispecific Antibody Biopolymer Conjugate (ABC) / OphthalmologyWet Age-Related Macular Degeneration (wet AMD)Diabetic Macular Edema (DME)
- KSI-101KSI-101 / IL-6 / VEGF / Bispecific Protein / OphthalmologyMacular Edema Secondary to Inflammation (MESI)
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- mivocabtagene autoleucelKYV-101 / CD19 / Autologous CAR T-cell therapy / Autoimmune DiseasesStiff 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 DiseasesAutoimmune DiseasesPhase 1IND ActiveUS
- KYV-201KYV-201 / CD19 / Allogeneic CAR T-cell therapy / Autoimmune DiseasesAutoimmune DiseasesPreclinicalActiveUS
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- SotagliflozinINPEFA / ZYNQUISTA / SGLT1 / SGLT2 / Small Molecule / CardiometabolicHeart FailureApprovedactiveUnited StatesHypertrophic Cardiomyopathy (HCM)Phase 3activeGlobalType 1 Diabetes (T1D)RegistrationactiveUnited States
- PilavapadinLX9211 / AAK1 / Small Molecule / Pain / NeurologyDiabetic Peripheral Neuropathic Pain (DPNP)Phase 2bactiveUnited StatesPostherpetic Neuralgia (PHN)Phase 2inactiveUnited States
- LX9851LX9851 / ACSL5 / Small Molecule / MetabolismObesity / Weight ManagementPhase 1activeGlobal
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- ZidesamtinibNVL-520
- NeladalkibNVL-655
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- LicaminlimabOCS-02
- PrivosegtorOCS-05
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- D-PLEX100D-PLEX100 / Bacterial Ribosome / Small Molecule Formulation / Infectious Diseases / Surgical Site CarePrevention of surgical site infections (SSIs) following abdominal colorectal surgeryPhase 3OngoingUnited StatesEuropeIsrael
- PP03APP03A / GLP-1 Receptor / Peptide Formulation / Metabolic DiseaseObesity and Type 2 DiabetesPreclinicalOngoing
- PP04APP04A / OncoPLEX / STING / Small Molecule Formulation / OncologySolid TumorsPreclinicalOngoing
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- QRX003QRX003 / Serine protease inhibitor / Small molecule / DermatologyNetherton SyndromePhase 3OngoingUnited StatesEuropeMiddle EastPeeling Skin SyndromePhase 2OngoingNew Zealand
- Topical rapamycinQRX009 / mTOR / Small molecule / DermatologyMicrocystic Lymphatic MalformationsPreclinicalOngoingGlobalVenous MalformationsPreclinicalOngoingGlobalAngiofibromaPreclinicalOngoingGlobal
- QRX008QRX008 / VLA-4 / VCAM-1 inhibitor / Small molecule / DermatologySclerodermaPreclinicalOngoingGlobal
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- RAP-219RAP-219 / TARPγ8 AMPAR / Small molecule / Neurology/PsychiatryFocal onset seizuresPrimary generalized tonic-clonic seizuresBipolar mania
- α6β4 nAChR Agonistα6β4 nAChR / Small molecule / NeurologyChronic painMigraine
- α9α10 nAChR Modulatorα9α10 nAChR / Small molecule / Hearing/VestibularHearing disordersVestibular disorders
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- PC111PC111 / sFasL (soluble Fas Ligand) / Monoclonal Antibody / Immunology / DermatologyPemphigusPreclinicalActiveGlobalStevens-Johnson syndrome / toxic epidermal necrolysis (SJS/TEN)PreclinicalActiveGlobal
- Intradermal IL-17A/F NanoAbIL-17A / IL-17F / VHH Antibody Fragment (NanoAb) / Immunology / DermatologyMild-to-moderate plaque psoriasisPreclinicalActiveGlobal
- Systemic Bi-Specific IL-17 NanoAbIL-17A / IL-17F and secondary proprietary target / Bi-specific VHH Antibody Fragment (NanoAb) / Immunology / DermatologyModerate-to-severe plaque psoriasisPreclinicalActiveGlobalPsoriatic arthritisPreclinicalActiveGlobal
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- DenifanstatASC40 / FASN / Small molecule / Metabolic disease, Dermatology, OncologyMASH (F2/F3)Phase 3ActiveModerate to severe acnePhase 3ActiveRecurrent glioblastoma (GBM)Phase 3Active
- TVB-3567FASN / Small molecule / Dermatology, OncologyAcnePhase 1ActiveSolid tumorsPreclinicalActive
- FASNi TopicalFASN / Small molecule / DermatologyAcnePreclinicalActive
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- lotilaner ophthalmic solution 0.25%TP-03 / XDEMVY / Small molecule / Eye CareDemodex blepharitisMeibomian Gland Disease
- lotilaner ophthalmic gelTP-04 / Small molecule / Dermatology / Eye CareRosaceaOcular rosacea
- lotilaner oral tabletTP-05 / Small molecule / Infectious Disease PreventionLyme disease preventionCommunity reduction of malaria
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- TX45TX-000045 / RXFP1 / Fc-relaxin fusion protein / CardiopulmonaryPH-HFpEF (Group 2 Pulmonary Hypertension)PH-HFrEF (Group 2 Pulmonary Hypertension)PH-ILD (Group 3 Pulmonary Hypertension)
- TX2100TX002100 / GPCR / Biotherapeutic / GPCR Antagonist / Hematology / Rare DiseasesHereditary Hemorrhagic Telangiectasia (HHT)
- Fibrosis ProgramGPCR / Bispecific GPCR Antagonist / FibrosisFibrosis
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- ublituximabTG-1101 / BRIUMVI / CD20 / Monoclonal Antibody / Autoimmune DiseasesRelapsing Multiple Sclerosis (RMS)
- ublituximab subcutaneousCD20 / Monoclonal Antibody / Autoimmune DiseasesRelapsing Multiple Sclerosis (RMS)
- azercabtagene zapreleucelazer-cel / CD19 / Allogeneic CAR-T Cell Therapy / Autoimmune DiseasesProgressive Multiple Sclerosis (PMS)Autoimmune Diseases
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- Intranasal ForalumabTZLS-401 / CD3 epsilon / Monoclonal Antibody / Neurology/ImmunologyNon-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 / OncologyNSCLC with pan KRAS-positive mutations
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- TN-201TN-201 / MYBPC3 / AAV-based gene therapy / CardiovascularMYBPC3-associated Hypertrophic Cardiomyopathy (HCM)
- TN-401TN-401 / PKP2 / AAV9-based gene therapy / CardiovascularPKP2-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
- TN-301TN-301 / HDAC6 / Small Molecule / Cardiometabolic / NeuromuscularHeart Failure with preserved Ejection Fraction (HFpEF)Duchenne Muscular Dystrophy (DMD)
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- sparsentanFILSPARI
- pegtibatinaseTVT-058
- tioproninThiola / Thiola EC
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- dabogratinibTYRA-300 / FGFR3 / Small molecule / Oncology / Skeletal DysplasiaMetastatic Urothelial Cancer (mUC)Non-Muscle Invasive Bladder Cancer (IR NMIBC)Achondroplasia
- TYRA-200TYRA-200 / FGFR1/2/3 / Small molecule / OncologyIntrahepatic CholangiocarcinomaAdvanced Solid Tumors
- TYRA-430TYRA-430 / FGFR4/3 / Small molecule / OncologyHepatocellular Carcinoma (HCC)Advanced Solid Tumors
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- AtaciceptVT-001 / BAFF and APRIL / Recombinant Fusion Protein / Immunology / NephrologyIgA Nephropathy (IgAN)BLA SubmittedPriority Review (PDUFA July 2026)USGlobalLupus NephritisPhase 2OngoingGlobalFocal Segmental Glomerulosclerosis (FSGS)Phase 2OngoingGlobal
- MAU868MAU868 / BK Virus / Monoclonal Antibody / Infectious Disease / TransplantationBK Viremia in Kidney Transplant RecipientsPhase 2Evaluating for future developmentGlobal
- VT-109VT-109 / BAFF and APRIL / Fusion Protein / ImmunologyB Cell Mediated DiseasesPreclinicalActiveGlobal
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- Avutometinib and DefactinibVS-6766 / VS-6063 / AVMAPKI FAKZYNJA CO-PACK / RAF/MEK and FAK / Small Molecule / OncologyKRAS-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 / OncologyBRAF, KRAS and NRAS-Mutant Solid TumorsPhase 1ActiveGlobal
- VS-7375GFH375 / KRAS G12D / Small Molecule / OncologyKRAS 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 DiseasesHidradenitis suppurativaSystemic sclerosis
- crebankitugZB-168 / IL-7Rα / Monoclonal antibody / Autoimmune and Inflammatory DiseasesAutoimmune and inflammatory conditions
- torudokimabZB-880 / IL-33 / Monoclonal antibody / Autoimmune and Inflammatory DiseasesAutoimmune and inflammatory conditions
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- ImdusiranAB-729; IDR / HBV viral antigens/HBV RNA transcripts / GalNAc-conjugated RNAi therapeutic; subcutaneous / Infectious disease; hepatitis BChronic 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 BChronic 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 diseasesLupus nephritisPhase 1activeSystemic lupus erythematosusPhase 1activeSystemic sclerosisPhase 1active
- ADI-212ADI-212 / PSMA / Next-generation gene-edited and armored allogeneic CAR gamma delta T cell therapy / OncologyMetastatic castration-resistant prostate cancerPreclinicalactive
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- AKY-1189AKY-1189 / Nectin-4 / miniprotein radioconjugate; 225Ac targeted alpha radiopharmaceutical / OncologyNectin-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 / OncologyMetastatic 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 / OncologyTROP2-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 / OncologyCEACAM5-expressing solid tumorsDiscoveryAdvancing
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- evorpaceptALX148 / CD47 / CD47-targeted investigational therapy / OncologyENHERTU-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 / OncologyEGFR-expressing solid tumorsPhase 1FPI mid-year 2025
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- AL001AL001 / LISPRO / ionic cocrystal of lithium, proline and salicylate / Neurology/PsychiatryAlzheimer'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 / NeurologyMild to moderate dementia of the Alzheimer's typePhase 1/2apaused in 2024; expected to resume by mid-2026US
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- dostarlimabJemperli / PD-1 / antibody / OncologyEndometrial cancerApprovedapproveddMMR pan tumorsApprovedapprovedRectal cancerbeing studied
- imsidolimabIL-36R / antibody / Dermatology/InflammationGeneralized pustular psoriasisBLA submitted/acceptedFDA target action date December 12, 2026US
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- EpetraboroleLeucyl-tRNA synthetase / oral boron-based small molecule antibiotic / Hematology; Infectious diseasePhlebotomy-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 diseaseChagas diseasePhase 1first-in-human trial underway
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- bemnifosbuvir/ruzasvir fixed-dose combinationBEM/RZR / HCV NS5B polymerase; HCV NS5A / oral small-molecule antiviral regimen / viral hepatitishepatitis C virus (HCV) infectionPhase 3activeglobal
- AT-587AT-587 / hepatitis E virus (HEV) / oral nucleotide analog / viral hepatitischronic hepatitis E virus (HEV) infection in immunocompromised patientspreclinicalPhase 1 anticipated mid-2026
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- LarsucosterolDUR-928 / Epigenetic modulator / HepatologyAlcohol-associated hepatitisPhase 3Registrational Phase 3 initiated in early 2026US
- AmiselimodMT-1303 / S1P receptor / Oral S1P modulator / GastroenterologyMild-to-moderate ulcerative colitisPhase 2Internal review of opportunity ongoing
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- BezisterimNE3107 / ERK; NF-κB; TNF-α / oral small molecule anti-inflammatory insulin sensitizer / neurology; neurodegenerative diseaseAlzheimer’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 diseaseascites 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 / OncologyGlioblastomaPhase 1Other cancersPreclinical
- MotixafortideBL-8040 / APHEXDA / CXCR4 / peptide CXCR4 inhibitor / Oncology; rare diseases; stem cell mobilizationPancreatic cancerPhase 2Stem cell mobilization in multiple myelomaApprovedApproved in US; bridging study in AsiaUSAsiaSickle cell disease stem cell mobilizationPhase 1/2
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- TinlarebantLBS-008 / RBP4 / Oral small-molecule RBP4 antagonist / Ophthalmology / retinal degenerative diseasesStargardt 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 diseaseNon-alcoholic fatty liver disease (NAFLD)PreclinicalPreclinical developmentNon-alcoholic steatohepatitis (NASH)PreclinicalPreclinical developmentType 2 diabetes (T2D)PreclinicalPreclinical development
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- IcovamenibMenin / Oral small molecule menin inhibitor / Diabetes; metabolic diseaseType 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 diseaseHealthy participants / obesity and metabolic disordersPhase IOngoing
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- CAN-2409CAN-2409 / Adenovirus platform viral immunotherapy / OncologyBorderline 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 / OncologyRecurrent high-grade glioma / recurrent GBMPhase IFast Track Designation (FDA)US
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- BarzolvolimabCDX-0159 / KIT / KIT antagonist mAb / Immunology/inflammationChronic spontaneous urticariaPhase 3ongoingCold urticariaPhase 3ongoingSymptomatic dermographismPhase 3ongoing
- CDX-622CDX-622 / TSLP; SCF / Bispecific antibody / Immunology/inflammationHealthy volunteersPhase 1ongoingAsthma proof of mechanismPhase 1ongoing
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- COMP360COMP360 / synthetic psilocybin treatment / Mental healthTreatment-resistant depressionPhase 3NDA rolling submission underwayUSUKPost-traumatic stress disorderPhase 2b/3IND accepted; trial initiation enabledUSAnorexia nervosaPhase 2
- Prodrug programmeprodrug programme
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- CRB-701CRB-701 / Nectin-4 / antibody-drug conjugate / OncologyNectin-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 diseaseObesity and related conditionsPhase 1 in progress12-week dose-range study in obesity planned mid-2026
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- Vispacabtagene regedleucelCB-010 / vispa-cel / CD19 / Allogeneic anti-CD19 CAR-T cell therapy; PD-1 knockout / Hematologic malignanciesRelapsed 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 malignanciesRelapsed or refractory multiple myelomaPhase 1Ongoing
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- VIASKIN® Peanut PatchDBV712 / Peanut allergen extract / Epicutaneous immunotherapy patch; drug-device combination / Food allergyPeanut 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 / immunologyCow’s milk allergy; ages 2–17 yearsPhase 2MILES study referencedMilk-induced eosinophilic esophagitis; ages 4–17 yearsPhase 2SMILEE study referenced
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- DT120 ODTDT120 / serotonin receptors / Orally disintegrating tablet formulation of lysergide (LSD) D-tartrate / Psychiatry / brain healthGeneralized 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 disordersAutism spectrum disorderPhase 2aPhase 2a study initiated after completion of Phase 1 single-ascending dose study
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- DM199 (rinvecalinase alfa)DM199 / KLK1 / Recombinant human tissue kallikrein-1 protein (rhKLK1) / NeurologicalAcute Ischemic StrokePhase 2/3ReMEDy2 Study; in progress
- DM199 (rinvecalinase alfa)DM199 / KLK1 / Recombinant human tissue kallikrein-1 protein (rhKLK1) / Pregnancy ComplicationsPreeclampsiaPhase 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
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- ClaseprubartDNTH103 / active C1s / long-acting monoclonal antibody / autoimmune / neuromuscular disordersGeneralized 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 disordersSystemic lupus erythematosus (SLE)Phase ITwo-part Phase 1 study in China ongoing; healthy-volunteer results expected 2H 2026China
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- PBGENE-HBVPBGENE-HBV / HBV cccDNA and integrated HBV DNA / in vivo ARCUS gene editing therapy / Infectious diseaseChronic 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 diseaseDuchenne muscular dystrophyPhase 1/2FUNCTION-DMD study; first clinical trial site activated and enrollment begun per company IR overviewUnited States
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- CDC Category A AgentCDC Category A agent / Medical countermeasuresUndisclosed Disease Area
- CDC Category B AgentCDC Category B agent / Medical countermeasuresUndisclosed Disease Area
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- EP-104GIEP-104GI / long-acting controlled-release fluticasone propionate formulation; intra-esophageal injection / Gastrointestinal / inflammatory diseaseEosinophilic esophagitis (EoE)Phase 1b/2Ongoing
- EP-104IAREP-104IAR / long-acting controlled-release fluticasone propionate formulation; intra-articular injection / Pain / osteoarthritisPain due to knee osteoarthritisPhase 2Completed
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- EVO756EVO756 / MRGPRX2 / Oral small-molecule antagonist / Chronic inflammatory diseasesChronic Spontaneous UrticariaPhase 2Atopic DermatitisPhase 2MigrainePhase 1
- EVO301EVO301 / IL-18 / Long-acting injectable SAFA-IL-18BP fusion protein / Chronic inflammatory diseasesAtopic DermatitisPhase 2Ulcerative ColitisPhase 1
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- DURAVYUEYP-1901 / DURAVYU / VEGF receptors; JAK1; PDGFR / vorolanib intravitreal insert; tyrosine kinase inhibitor in Durasert E bioerodible sustained-release insert / Retinal diseasesWet 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 diseasesRetinal diseasesPre-clinicalIn progress
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- FID-007FID-007 / Cytotoxic / Nanoencapsulated paclitaxel / OncologyHead 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 / OncologyColon cancerPhase 1Ongoing; dosing for 3rd dose level ongoingPancreatic cancerPhase 1Ongoing; dosing for 3rd dose level ongoingOvarian cancerPhase 1Ongoing; dosing for 3rd dose level ongoing
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- RevitaDuodenal mucosa / Endoscopic hydrothermal ablation / ObesityWeight 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 DiabetesType 2 diabetesPhase 1/2CTA submitted; FIH dosing & preliminary data expected H2 2026 pending approvalEUAustralia
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- BatoclimabIMVT-1401
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- FG-3246FOR46 / CD46 / Antibody-Drug Conjugate (ADC) / OncologyMetastatic Castration-Resistant Prostate Cancer (mCRPC)Phase 2OngoingUnited States
- roxadustatFG-4592 / HIF-PH / Small Molecule / Rare DiseaseAnemia associated with lower-risk myelodysplastic syndromes (LR-MDS)Phase 3PlannedUnited States
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- rondecabtagene autoleucelLYL314 / Ronde-cel / CD19 / CD20 / CAR T-cell therapy / OncologyRelapsed/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 / OncologyRefractory Metastatic Colorectal Cancer (mCRC)Phase 1ActiveUS
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- MT-601neldaleucel / Survivin, PRAME, NY-ESO-1, MAGE-A4, SSX2, WT-1 / T Cell Therapy (MAR-T cell) / OncologyLymphomaPancreatic Cancer
- MT-401-OTSzelenoleucel / Multi-Tumor Associated Antigens / Allogeneic T Cell Therapy / OncologyAcute Myeloid Leukemia (AML)Myelodysplastic Syndrome (MDS)
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- ObicetrapibAMG-899 / CETP / Small Molecule / CardiometabolicPrimary hypercholesterolemia (HeFH and non-familial or mixed dyslipidemia)RegistrationOngoingGlobalEuropeAtherosclerotic Cardiovascular Disease (ASCVD)Phase 3OngoingGlobal
- Obicetrapib / Ezetimibe FDCCETP / NPC1L1 / Small Molecule / CardiometabolicPrimary hypercholesterolemia (HeFH and non-familial or mixed dyslipidemia)RegistrationOngoingGlobalEurope
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- NRX-101NMDA and 5-HT2A receptors / Small molecule / Central Nervous SystemBipolar 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 SystemAcute Suicidal Depression / Suicidal Ideation in DepressionRegistrationActiveUnited States
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- RinzimetostatORIC-944 / PRC2 (EED subunit) / Small molecule / OncologyMetastatic Castration-Resistant Prostate Cancer (mCRPC)Phase 1bActiveGlobal
- EnozertinibORIC-114 / EGFR / HER2 (exon 20 and PACC/atypical mutations) / Small molecule / OncologyNon-Small Cell Lung Cancer (NSCLC)Phase 1bActiveUnited StatesAustraliaCanadaHong KongMalaysiaPolandSouth KoreaSpainTaiwanUnited Kingdom
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- rhenium Re186 obisbemeda186RNL / REYOBIQ / Radiotherapeutic / OncologyRecurrent GlioblastomaLeptomeningeal MetastasesPediatric Brain Cancer
- Rhenium-188 NanoLiposome Biodegradable Alginate Microsphere188RNL-BAM / Radiotherapeutic / OncologyHepatocellular CarcinomaMetastatic Colorectal Cancer
2assets
- Micvotabart pelidotinMICVO, PYX-201 / EDB+FN / Antibody-Drug Conjugate (ADC) / OncologySolid TumorsRecurrent and Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)
- PYX-106PYX-106 / Siglec-15 / Monoclonal Antibody (mAb) / OncologySolid Tumors
2assets
2assets
- BempikibartADX-914
2assets
- vusolimogene oderparepvecRP1 / HSV-1 oncolytic virus encoding GALV-GP R-protein and GM-CSF / Oncolytic Virus / OncologyAdvanced MelanomaPhase 3Complete Response LetterUSNon-melanoma Skin Cancers (CSCC, MCC, BCC, Angiosarcoma)Phase 2ActiveUS
- RP2RP2 / Oncolytic virus / Oncolytic Virus / OncologyMetastatic Uveal MelanomaPhase 2ActiveUSHepatocellular CarcinomaPhase 2ActiveUS
2assets
2assets
2assets
- BIM-IOL SystemBimatoprost Drug Pad-IOL System
- BIM-DRSBimatoprost Drug Ring System
2assets
- SLS009tambiciclib
2assets
- RevumenibSNDX-5613 / Revuforj / Menin / Small Molecule / OncologyR/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 / ImmunologyRefractory 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 DiseaseMucopolysaccharidosis Type IIIB (MPS IIIB) / Sanfilippo Syndrome Type BPhase 3ActiveGlobal
- TildacerfontCorticotropin-releasing factor type-1 (CRF1) receptor / Small Molecule / Neurology / PsychiatryMajor Depressive Disorder (MDD)Phase 2ActiveEurope
2assets
- SL-325SL-325 / DR3 (Death Receptor 3) / Monoclonal Antibody / Inflammatory and Immune-Mediated DiseasesInflammatory Bowel Disease (IBD)Crohn's DiseaseUlcerative Colitis
- SL-425SL-425 / DR3 (Death Receptor 3) / Monoclonal Antibody / Inflammatory and Immune-Mediated DiseasesInflammatory Bowel Disease (IBD)
2assets
- TARA-002TARA-002 / Broad immunopotentiator / Cell therapy / Oncology / Rare DiseasesNon-Muscle Invasive Bladder Cancer (NMIBC)Phase 2ActiveUnited StatesLymphatic Malformations (LMs)Phase 2ActiveUnited States
- IV Choline ChloridePhospholipid substrate replacement / Small molecule / Intravenous replacement therapy / Rare DiseasesPatients dependent on long-term parenteral support (PS) / Choline deficiencyPhase 3ActiveUnited States
2assets
2assets
- Oxylanthanum CarbonateOLC / Renazorb / Phosphate / Small molecule / NephrologyHyperphosphatemia in chronic kidney disease (CKD) on dialysisNDAResubmittedUnited States
- UNI-494UNI-494 / KATP channel / Nitric oxide (nicorandil prodrug) / Small molecule / NephrologyAcute Kidney Injury (AKI)Phase 1CompletedUnited KingdomUnited StatesPrevention of Delayed Graft Function (DGF) in kidney transplant patientsPhase 1ActiveUnited States
2assets
- EnobosarmVERU-024 / GTx-024 / Ostarine
- SabizabulinVERU-111
2assets
- cantharidinVP-102 / YCANTH / Unknown / Small Molecule / DermatologyMolluscum ContagiosumApprovedActiveCommon WartsPhase 3ActiveExternal Genital WartsPhase 2Active
- VP-315VP-315 / Oncolytic peptide / Peptide / Dermatologic OncologyBasal Cell CarcinomaPhase 2ActiveSquamous Cell CarcinomaPhase 1/2Active
2assets
- VAR 200Cholesterol Efflux Pathway (ABCA1 and ABCG1) / Cholesterol Efflux Mediator / Renal DiseasesFocal Segmental Glomerulosclerosis (FSGS)Phase 2activeUnited StatesDiabetic Kidney Disease (DKD)Phase 2activeUnited StatesAlport SyndromePreclinicalactiveUnited States
- IC 100Inflammasome ASC / Monoclonal Antibody / Inflammatory DiseasesCardiometabolic Conditions (Obesity-associated)PreclinicalactiveUnited StatesParkinson's DiseasePreclinicalactiveUnited StatesOrphan Renal DiseasesPreclinicalactiveUnited States
1assets
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1assets
- cytisiniclinenicotinic acetylcholine receptor / plant-derived alkaloid; partial agonist / nicotine dependencesmoking 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 / Oncology1L 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 / OncologyLOTIS-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 disordersEpisodic migrainePhase 2United StatesChronic migrainePhase 2United StatesCervical dystoniaPhase 2United States
1assets
- Ampligenrintatolimod / Ampligen / TLR3 / dsRNA immuno-modulator / Oncology; immune disorders; viral diseasesMetastatic 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 diseasesMetabolic 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 diseasesMetastatic 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) / oncologyearly-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 diseasesHidradenitis 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 / Oncology1L 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 / DermatologyNew 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; neuromuscularOculopharyngeal muscular dystrophy (OPMD)Phase 1b/2aClinical-stage
1assets
- rese-celCABA-201 / CD19 / Autologous fully human 4-1BB CD19-CAR T cell therapy / Autoimmune diseaseDermatomyositisRegistrationalrecruitingUSAntisynthetase syndromeRegistrationalrecruitingUSImmune-mediated necrotizing myopathyPhase 1/2recruitingUS
1assets
- gedatolisibPI3K/AKT/mTOR pathway; class I PI3K isoforms; mTORC1/2 / small molecule kinase inhibitor / OncologyHR+/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 cancerHigh-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 diseasesAcute exacerbations of COPDPhase 2ongoingGlobalAcute exacerbations of asthmaPhase 2ongoingGlobalChronic asthmaongoing studies by SimcereChina
1assets
- onvansertibPLK1 / oral small-molecule PLK1 inhibitor / Oncology1L 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 diseasesHyperlipidemia / 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 diseasesCeliac 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 careReduce 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 / HematologySickle cell diseasePhase 1bactive; potential registration-enabling trial planned for 2H 2026United States
1assets
- imetelstatRYTELO / telomerase / telomerase inhibitor / Hematologic malignanciesJAK 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 / OncologyPrevention 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 / OncologyPlatinum-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 / HematologyRelapsed/refractory AL AmyloidosisPhase 2BLA-enabling; enrollment completedUSIsraelSelect immune-mediated diseasesExpanding
1assets
- TH103TH103 / VEGF / HSPG / Recombinant fusion protein / Retinal DiseasesNeovascular age-related macular degeneration (nAMD)Phase 1b/2OngoingDiabetic eye diseasePlannedPlannedRetinal vein occlusionPlannedPlanned
1assets
- Lomecel-Blaromestrocel
1assets
- LB-100LB-100 / Protein Phosphatase 2A (PP2A) / Small Molecule / OncologyOvarian Clear Cell CarcinomaPhase 2ActiveUnited StatesMetastatic Colon CancerPhase 1bActiveGlobalAdvanced Soft Tissue SarcomaPhase 1/2PausedEurope
1assets
- nomlabofuspCTI-1601
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 SyndromeUncontrolled 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 / OncologyLocally 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 DiseasesLupus NephritisSystemic SclerosisInflammatory Myopathy
1assets
- Tumor Treating FieldsTTFields / Optune Gio, Optune Lua, Optune Pax, Optune Mya / Mitotic spindle / Electric Field Therapy / OncologyGlioblastoma (GBM)ApprovedMarketedUnited StatesEuropeJapanGlioblastoma (GBM) - Earlier use (TRIDENT)Phase 3ActiveGlobalNon-Small Cell Lung Cancer (NSCLC)ApprovedMarketedUnited StatesJapanEurope
1assets
- PALI-2108PALI-2108 / PDE4 / Small Molecule Prodrug / GastroenterologyUlcerative Colitis (UC)Phase 2ActiveGlobalCrohn's Disease (CD)Phase 2ActiveGlobal
1assets
- DeucrictibantPHA121
1assets
- RezatapoptPC14586
1assets
- RilparencelREACT
1assets
- NimacimabCB1 / Monoclonal antibody / Metabolic DiseasesObesityPhase 2activeUnited States
1assets
- IvonescimabSMT112 / AK112
1assets
- Epinephrine nasal sprayARS-1 / neffy / Alpha and beta-adrenergic receptors / Small Molecule / Immunology & AllergyType 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 DiseasesAutoimmune Pulmonary Alveolar Proteinosis (aPAP)SubmissionRegulatory SubmissionUSUKEU
1assets
- TERN-701TERN-701 / BCR::ABL1 / Small Molecule / OncologyChronic Myeloid Leukemia (CML)Phase 1/2ActiveGlobal
1assets
- pacibekitugTOUR006 / IL-6 / Monoclonal Antibody / Cardiovascular and Immune DiseasesAtherosclerotic Cardiovascular Disease (ASCVD)Phase 2ActiveGlobalThyroid Eye Disease (TED)Phase 2ActiveGlobalAbdominal Aortic Aneurysm (AAA)Phase 2PlannedGlobal
1assets
- idecabtagene vicleucelAbecma / BCMA / CAR-T Cell Therapy / OncologyRelapsed or refractory multiple myelomaMarketedApprovedUSEU
1assets
- verekitugUPB-101 / TSLP receptor (TSLPR) / Monoclonal antibody / Respiratory/Inflammatory DiseasesSevere AsthmaChronic rhinosinusitis with nasal polyps (CRSwNP)Chronic obstructive pulmonary disease (COPD)
1assets
- TelitaciceptRC18 / Taiai / BAFF/APRIL (BLyS/APRIL) / Recombinant Fusion Protein / Autoimmune DiseasesGeneralized 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 / EndocrinologyType 1 Diabetes (adjunctive to insulin)Phase 3OngoingUnited StatesType 2 DiabetesPhase 2PlannedUnited Arab Emirates
1assets
- AzenosertibZN-c3 / WEE1 / Small Molecule / OncologyCyclin E1-positive platinum-resistant ovarian cancer (PROC)Advanced solid tumors
Adaptive Biotechnologies Corporation
No pipeline0assets
Adaptive Biotechnologies Corporation
No pipelineNo disclosed assets in this row.