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Beam Therapeutics Inc.

Pipeline6 assets

  • Ristoglogene autogetemcelBEAM-101 / Risto-cel / HBG1/2 promoter; fetal hemoglobin activation / Ex vivo autologous CD34+ HSPC base-edited cell therapy / Hematology
    Sickle cell diseasePhase I/IIOngoing BEACON Phase 1/2 trial; FDA orphan drug and RMAT designationsU.S.
  • ESCAPEBEAM-103 / CD117; HbF edit + CD117 edit-antibody pair / Anti-CD117 monoclonal antibody plus edited cell therapy; in vivo and/or ex vivo / Hematology
    Sickle cell diseasePhase I-EnablingBeta-thalassemiaPhase I-Enabling
  • In vivo HSC editingHbF activation / In vivo LNP base editing / Hematology
    Sickle cell diseaseResearchBeta-thalassemiaResearch
  • BEAM-302BEAM-302 / SERPINA1 E342K/PiZ mutation correction / In vivo LNP base editing / Liver-targeted genetic diseases
    Alpha-1 antitrypsin deficiencyPhase I/IIOngoing Phase 1/2 trial; FDA orphan drug and RMAT designationsU.S.
  • BEAM-301BEAM-301 / G6PC R83C mutation correction / In vivo LNP base editing / Liver-targeted genetic diseases
    Glycogen storage disease IaPhase I/IIOngoing Phase 1/2 trial
  • BEAM-304BEAM-304 / PAH mutation correction / In vivo LNP base editing / Liver-targeted genetic diseases
    PhenylketonuriaPhase I-EnablingPlanned Phase 1/2 trialU.S.

Latest news12 items

All news

Beam Therapeutics Presents Recently Reported Topline Clinical Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at the American Thoracic Society (ATS) 2026 International Conference

Presentation Features Additional Data from the Single-dose Cohorts of the Phase 1/2 Trial, Including Detailed Safety Results, Efficacy Durability and Reduction in Human Neutrophil Elastase Activity Post-BEAM-302 Treatment CAMBRIDGE, Mass., May 18, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc.

Presentation / Abstract • Phase 1/2ATS symposium presentation Mon, May 18, 2026, 4:00 PM EDT

Beam Therapeutics to Present Updated Biomarker Data from Phase 1/2 BEACON Trial Further Underscoring Risto-cel’s Ability to Restore Red Blood Cell Health and Function in Sickle Cell Disease at EHA2026

CAMBRIDGE, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present updated biomarker data from the BEACON Phase 1/2 clinical trial of

Presentation / Abstract • Phase 1/2Poster presentation Sat, Jun 13, 2026, 6:45 PM CEST

Beam Therapeutics Announces Compelling Updated Clinical Data from the Ongoing Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) to Support Advancement to Pivotal Development

Treatment with 60 mg of BEAM-302 Led to Mean Steady-state Total AAT Level of 16.1 µM and All Patients Consistently and Durably Above the 11 µM Protective AAT Threshold with up to 12 Months of Follow-up Corrected M-AAT Comprised 94% of Total AAT with a Concomitant 84% Reduction in Mutant Z-AAT

Trial Results • Phase 1/2

Beam Therapeutics Sets Strategic Priorities for its Genetic Disease and Hematology Franchises to Drive Execution of Late-Stage Clinical Programs and Extends its Operating Runway through Commercial Transition

Alignment Reached with U.S. FDA on Potential Accelerated Approval Pathway for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) Based on Biomarker Endpoints U.S. B iologics Licensing Application (BLA) Submission for risto-cel (Previously Known as BEAM-101) Expected as Early as Year-End 2026

Beam Therapeutics Reports Updated Data from BEACON Phase 1/2 Trial of ristoglogene autogetemcel (risto-cel) Highlighting Durable, Differentiated Profile in Sickle Cell Disease (SCD) at American Society of Hematology (ASH) Annual Meeting

Updated Data from 31 Adult and Adolescent SCD Patients Treated with risto-cel (Formerly BEAM-101) Show Mean He moglobin F (HbF) Induction of >60%, Hemoglobin S (HbS) Reduction to 40%, and Resolution of Anemia Durable for up to 20 Months Patients Required a Median of One Cell Collection Cycle and

Presentation / Abstract • Phase 1/2
Beam Therapeutics Inc. • Endpoint Arena