Biopharma Transcript

Beam Therapeutics Inc. BEAM

Nasdaq · Phase 3 · hematology · ex vivo base editing · EDGAR · Updated Nov 5, 2026

Beam Therapeutics Inc. (Nasdaq: BEAM) is a hematology drug developer. Its lead program, risto-cel, is in Phase 3 for sickle cell disease. Its next catalyst on record is risto-cel: Updated data from BEACON trial (2026).

Upcoming catalysts

No catalyst is dated to the next 30 days or to an FDA action date.

Next further out: risto-cel — Updated data from BEACON trial (2026). 4 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.

Pipeline

ProgramIndicationModalityStage
risto-celsickle cell diseaseex vivo base editingPhase 3
BEAM-302alpha-1 antitrypsin deficiency (AATD)in vivo LNP base editingPhase 2
BEAM-301glycogen storage disease Ia (GSDIa)in vivo LNP base editingPhase 2
BEAM-304phenylketonuria (PKU)in vivo LNP base editingPhase 1
BEAM-103anti-CD117 antibody for ESCAPE platformantibodyPhase 1
In vivo HSC-targeted LNPsickle cell diseasein vivo LNP base editingPre-clinical

Recent results and decisions

  1. Nov 3, 2026
    n/a U.S. BLA submission for risto-cel in sickle cell disease expected as early as year-end 2026.Beam expects to report updated clinical data by year-end 2026 and submit a U.S. biologics license application (BLA) for risto-cel as early as year-end 2026. Filing
  2. Nov 3, 2026
    n/a Dosing in Phase 1/2 trial of BEAM-304 in PKU anticipated to initiate in early 2027.Beam expects to initiate dosing in the Phase 1/2 trial in early 2027. Filing
  3. Oct 31, 2026
    positive Initial BEAM-301 data showed dose-dependent improvements in time to fasting hypoglycemia with two of three patients in the 40 mg cohort achieving maximum controlled fasting duration of ≥15 hours and complete elimination of starch supplementation.Initial BEAM-301 clinical data demonstrated dose-dependent improvements in time to fasting hypoglycemia (TTH) across all patients, with two of three patients in the 40 mg cohort achieving the protocol-defined maximum controlled fasting duration of ≥15 hours, consistent with restored endogenous gluco Filing

3 more on record.

For subscribers

Other phase 3 hematology companies