Beam Therapeutics reports initial Phase 1/2 data for BEAM-301 in glycogen storage disease type Ia trial
BEAM•Beam Therapeutics reported initial Phase 1/2 results for BEAM-301, showing signs that patients could maintain glucose levels after a single dose, with stronger effects at higher doses. Patients reduced reliance on frequent starch dosing; no dose-limiting toxicities or deaths were reported.
1. Initial trial findings
Beam Therapeutics said initial Phase 1/2 results for BEAM-301 in glycogen storage disease type Ia showed signs of restored ability to maintain glucose levels following a single dose, with stronger effects at higher dosing. Patients also reduced reliance on frequent starch dosing, supporting early clinical proof of concept for mutation-targeted genetic correction.
2. Safety and presentation
The safety profile appeared consistent with lipid nanoparticle therapies, with manageable infusion reactions and no dose-limiting toxicities or deaths reported. The company scheduled an investor webcast for Oct. 27, 2026, ahead of a poster presentation on Oct. 28 at ESGCT.
