Beam Therapeutics announced positive initial data from its Phase 1/2 trial of BEAM-301, an investigational base-editing therapy for glycogen storage disease type Ia (GSDIa). The data will be presented at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress and showed clinical proof-of-concept for correcting the disease-causing R83C mutation.
BEAM-301 produced dose-dependent improvements in key disease markers. In the higher-dose cohort, evaluable patients were able to fast for 15 hours or more, a significant improvement from baseline. They also had substantial reductions in their daily cornstarch needs to manage hypoglycemia.
The therapy’s safety profile was deemed acceptable and consistent with similar LNP-based treatments. Reported adverse events, including infusion-related reactions, resolved.