Beam Therapeutics Q1 2026: BEAM-302 AATD Pivotal Cohort Set for H2 2026, Risto-cel BLA Targeted Year-End; Cash Runway Extended to Mid-2029

Beam Therapeutics reported Q1 2026 financial results and pipeline milestones on May 7, 2026, marking a pivotal period for base-editing therapeutics. BEAM-302 for alpha-1 antitrypsin deficiency (AATD): following the March 2026 announcement of compelling updated Phase 1/2 data (29 patients treated, 60 mg selected as optimal biological dose producing mean steady-state total AAT of 16.1 µM, >94% M-AAT composition, ~84% Z-AAT reduction, all patients above the 11 µM protective threshold), Beam confirmed the global pivotal cohort is expected to initiate in H2 2026, targeting ~50 additional patients for BLA support. Risto-cel (rizotasgene autotemcel; ex vivo base-edited SCD cell therapy): following the April 1, 2026 NEJM publication of BEACON Phase 1/2 data showing zero severe vaso-occlusive crises in 31 treated patients and durable HbF >60%, the company is targeting U.S. BLA submission as early as year-end 2026. BEAM-304 (PKU, liver LNP base editing): IND application anticipated in 2026. BEAM-301 (GSDIa): data anticipated in 2026. Financial highlights: Q1 2026 revenue of $31.7M (up from $7.5M in Q1 2025), driven by $25.0M milestone from the Eli Lilly collaboration; R&D spend $104.5M; net loss narrowed to $94.3M ($0.91/share); cash, cash equivalents, and marketable securities of $1.2B (including $100M Sixth Street financing), with runway extended to mid-2029.