BEAM-302

August 4, 2026: Beam Therapeutics (NASDAQ: BEAM) provided a multi-program update. BEAM-302 (alpha-1 antitrypsin deficiency) dosed the first patient in the global pivotal cohort of its ongoing Phase 1/2 trial in July for AATD-associated lung disease, with or without liver disease; the cohort is intended to support a potential U.S. accelerated approval path. Updated BEAM-302 clinical data were selected for a late-breaking oral presentation at the ERS Congress on September 5-9, 2026.

BEAM-304 (phenylketonuria) received FDA IND clearance in June. Clinical start-up is underway for a planned Phase 1/2 trial initially assessing safety, tolerability, and reduction of blood phenylalanine in PKU patients with the R408W mutation, with later expansion to additional mutations. Updated preclinical data were presented in July at the FASEB Genome Engineering conference.

BEAM-301 (glycogen storage disease type Ia) remains in an open-label Phase 1/2 dose-exploration trial, with initial clinical data expected in 2026.

Risto-cel (formerly BEAM-101; sickle cell disease) completed dosing in all adult and adolescent patients in the Phase 1/2 BEACON trial. Beam expects updated BEACON data by year-end 2026 and a U.S. BLA submission as early as year-end 2026.

BEAM-103 completed Phase 1 healthy volunteer enrollment and dosing; treatment was well tolerated across all doses tested. Beam ended Q2 2026 with $1.2 billion in cash and projects runway into mid-2029.