Beam Therapeutics To Present Initial Data From Ongoing Phase 1/2 Clinical Trial Evaluating BEAM-301 For Treatment Of Glycogen Storage Disease Type Ia At 33rd Annual Congress Of ESGCT

Benzinga · 1d ago

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

Safety Profile of BEAM-301 Consistent with LNP-based Therapies

Beam to Host Investor Webcast on Tuesday, October 27, 2026, at 8:00 a.m. ET

CAMBRIDGE, Mass., Oct. 13, 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 initial data from the ongoing Phase 1/2 clinical trial evaluating BEAM-301 for the treatment of glycogen storage disease type Ia (GSDIa) at the 33rd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), taking place October 27-30, 2026, in Hamburg, Germany. BEAM-301 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the R83C mutation, one of the most prevalent disease-causing mutations in GSDIa and one that is associated with the most severe form of the disease.