First patient dosed in global pivotal cohort of BEAM-302

Beam Therapeutics/BEAM-302 (AATD)

Beam reported that in July 2026 it dosed the first patient in the global pivotal cohort of its BEAM-302 Phase 1/2 trial for alpha-1 antitrypsin deficiency (AATD), a one-time base-editing therapy that corrects the faulty gene. The pivotal cohort was designed to support a potential accelerated approval pathway in the US, following positive topline data showing 60 mg as the optimal dose.

• BEAM-302 is a liver-targeting base-editing therapy for alpha-1 antitrypsin deficiency
• 60 mg selected as optimal dose after Phase 1/2 data showed blood protein levels above protective threshold
• Global pivotal cohort of roughly 50 patients initiated in H2 2026 aiming for accelerated approval
• One-time infusion designed to permanently correct the PiZ mutation causing AATD