Beam Therapeutics Doses First Patient in Global Pivotal Cohort of BEAM-302 for AATD

Alongside its Q2 2026 financial results, Beam Therapeutics announced the first patient has been dosed in the global pivotal cohort of BEAM-302, its in vivo base-editing therapy correcting the Pi*Z mutation in SERPINA1 for alpha-1 antitrypsin deficiency (AATD) — currently the most advanced genetic medicine in development for the disease. Beam also completed dosing for all adult and adolescent sickle cell disease patients in the BEACON trial of risto-cel, and confirmed FDA IND clearance for BEAM-304, an in vivo base-editing therapy for phenylketonuria (PKU). BEAM-302 now faces competition from Prime Medicine's PM647, cleared to proceed in the same indication following the July 8 arbitration ruling.