BEAM Beam Therapeutics

BEAM-302 · NCT06389877 registry update

enrollment: 106 → 192; primary_outcomes: ['Phase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)', 'Phase 2 Dose Expansion: Absolute blood levels of total AAT'] → ['Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)', 'Absolute blood levels of total AAT', 'Mean total and functional AAT in blood']; countries: {'total_sites': 11, 'us_sites': 3} → {'total_sites': 12, 'us_sites': 3}

Beam Therapeutics doses first patient in BEAM-302 pivotal AATD cohort

Beam has dosed the first patient in the global pivotal cohort of the ongoing Phase 1/2 trial of BEAM-302 for AATD-associated lung disease, with or without liver disease. The cohort is designed to support a potential accelerated approval path in the United States. Updated clinical data for BEAM-302 were also selected for a late-breaking oral presentation at the ERS Congress 2026 (September 5-9, 2026).

Beam Therapeutics: BEAM-302 AATD topline data; pivotal cohort H2 2026

Updated Phase 1/2 topline data for BEAM-302 in AATD showed robust AAT increases and a well-tolerated profile, leading to selection of 60 mg as the optimal biological dose. FDA feedback supports an accelerated approval pathway, with a pivotal cohort of ~50 patients planned to start in the second half of 2026 to support a future BLA.

Beam Therapeutics plans risto-cel BLA submission as early as year-end 2026

The 8-K confirms that the company expects to submit a U.S. BLA for risto-cel in sickle cell disease as early as December 2026, supported by a new $500 million non-dilutive credit facility with Sixth Street that extends cash runway into mid-2029 and funds the anticipated commercial launch.