Beam Therapeutics Reports Initial Clinical Data from Phase 1/2 Trial of BEAM-301, Demonstrating Proof of Concept for Genetic Correction in Glycogen Storage Disease Type Ia (GSDIa) at ESGCT 33rd Annual Congress

Rebecca Riba-Wolman, Director of the glycogen storage disease program and disorders of hypoglycemia at Connecticut Children’s and University of Connecticut School of Medicine; presenting investigator in the BEAM-301 Phase 1/2 trial

“These findings demonstrate the potential for BEAM-301 to fundamentally change how patients with GSDIa manage their disease. The ability of two patients to achieve 15 hours of controlled fasting and no longer require starch supplementation following a single treatment represents a significant departure from the intensive daily management typically required for this disease. While these are early results, the improvements in glucose regulation and reductions in starch requirements provide encouraging evidence of BEAM-301’s potential to deliver meaningful and lasting benefits for patients.”

John Evans, Chief executive officer of Beam Therapeutics

“The promise of base editing is to deliver transformative outcomes that correct the root cause of disease with a one-time therapy. These initial results show that we are continuing to advance toward that vision, generating important clinical proof of concept for BEAM-301 in GSDIa and once again demonstrating the potential of base editing to fundamentally change the treatment paradigm for patients. These findings also reinforce the strength of our fully integrated platform, combining our proprietary base editing technology, validated delivery capabilities, and internal manufacturing to enable the predictable development and rapid production of precision genetic medicines. We look forward to building on these encouraging results as we advance BEAM-301 into the next dose cohort, engage with regulators on a potential pathway to approval, and progress our broader pipeline.”