Beam reports early data on gene therapy for rare metabolic disease
Beam Therapeutics Inc. (BEAM) reported initial data from its Phase 1/2 trial of BEAM-301 for glycogen storage disease type Ia. Two patients in the 40 mg cohort achieved 15 hours of controlled fasting. All patients experienced adverse events, with four having serious events. The company plans to engage with the FDA regarding a potential approval pathway.



