A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)

BEAM-301 is designed to correct the G6PC1 c.247C\>T allele via an A:T-to-G:C base-pair substitution, resulting in restoration of G6Pase-α catalytic activity.