FDA clears IND application for BEAM-301, first U.S. in vivo base editing program

Beam Therapeutics/BEAM-301

Beam received FDA clearance of the IND application for BEAM-301, its first regulatory filing for an in vivo base editing program. The company is advancing Phase 1/2 dose-exploration trial evaluating BEAM-301 for glycogen storage disease type Ia, with site activation commencing to support patient dosing in early 2025.

• BEAM-301 corrects R83C mutation, accounting for majority of GSDIa cases in most ethnic groups.
• LNP-delivered base editing targets liver cells, avoiding cell manufacturing needed for ex vivo therapies.
• First human program for Beam's in vivo base editing platform; Phase 1/2 includes safety, tolerability and efficacy readouts.