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GlobeNewswire·

Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causing Mutation

Single Dose of BEAM-302 Led to Durable, Dose-dependent Increases in Total and Functional Alpha-1 Antitrypsin (AAT), Production of Corrected M-AAT, and Decreases in Mutant Z-AAT in Circulation Across Initial Three Dose