Beam Therapeutics to Present Initial Data from Phase 1/2 Trial of BEAM-301 in Glycogen Storage Disease Type Ia (GSDIa) at the ESGCT 33rd Annual Congress
Globe Newswire•13/10/2026•07:00 ET
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Key Highlights
- ➤40 mg cohort achieved ≥15-hour controlled fasting by Day 36
- ➤BEAM-301 reduced prescribed daily starch intake up to 92%
- ➤Six patients received single infusions; three received 20 mg and three 40 mg
- ➤Five serious adverse events resolved within two to 10 days
- ➤No dose-limiting toxicities, discontinuations, or deaths were reported
Expert Statements
Amy Simon, Chief Medical Officer of Beam Therapeutics
“These initial data provide the first clinical evidence suggesting that BEAM-301, a base editing therapy designed to directly correct an underlying disease-causing mutation in patients with GSDIa, restored endogenous glucose production following a single in vivo treatment.”
Amy Simon, Chief Medical Officer of Beam Therapeutics
“Most notably, evaluable patients in the 40 mg cohort achieved ≥15 hours of controlled fasting and experienced substantial reductions in starch requirements.”
Amy Simon, Chief Medical Officer of Beam Therapeutics
“While follow-up remains ongoing, these findings support the transformative potential of BEAM-301 and represent an important proof of concept that precision genetic medicines, customized to a patient’s specific mutation, can address the root cause of their disease.”
BEAM-301 Achieves Clinical Proof of Concept for Genetic Correction of R83C Mutation in GSDIa
Initial Phase 1/2 Data Show Restoration of Endogenous Glucose Production Following a Single Treatment, with Dose-Dependent Improvements in Key GSDIa Endpoints
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