First patient dosed in BEAM-301 Phase 1/2 trial in glycogen storage disease type Ia

Beam Therapeutics/BEAM-301

Beam announced first patient dosed in the U.S.-based Phase 1/2 clinical trial evaluating BEAM-301 as potential treatment for patients with glycogen storage disease type Ia. The program is advancing dose-escalation with initial clinical data expected by year-end 2026.

• Phase 1/2 dose-exploration design includes sequential cohorts at increasing dose levels.
• R83C is the most common GSDIa mutation causing severe glucose metabolism defect.
• In vivo base editing avoids cell manufacturing and systemic delivery challenges of alternative approaches.