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Beam Therapeutics Inc. develops precision genetic medicines through a base-editing platform that combines gene editing, delivery and internal manufacturing capabilities. News about Beam Therapeutics centers on clinical and business updates for risto-cel in sickle cell disease, BEAM-302 in alpha-1 antitrypsin deficiency, liver-targeted genetic disease programs such as BEAM-304 in phenylketonuria and BEAM-301 in GSDIa, and financing activity tied to late-stage development.
Company updates also cover financial results, portfolio prioritization, published clinical data, FDA designations and regulatory interactions, licensing arrangements for base-editing patent rights, and governance changes.
Beam Therapeutics (Nasdaq: BEAM) reported second quarter 2026 net loss of $122.7 million ($1.18/share) on license and collaboration revenue of $0.5 million, with R&D expenses of $95.1 million and G&A expenses of $31.9 million. Cash, cash equivalents and marketable securities were $1.15 billion at June 30, 2026, and the company expects its cash plus an additional $200 million from its Sixth Street facility to fund operations into mid‑2029.
Operationally, Beam dosed the first patient in the global pivotal cohort of its Phase 1/2 BEAM‑302 trial in AATD, completed dosing of all adult and adolescent patients in the BEACON trial of risto‑cel in SCD, and began clinical start‑up for BEAM‑304 in PKU after U.S. IND clearance. Initial BEAM‑301 data in GSDIa and updated BEACON data are expected in 2026, with a potential risto‑cel BLA submission as early as year‑end 2026.
Beam Therapeutics (Nasdaq: BEAM) will present updated clinical data from its Phase 1/2 trial of BEAM-302 for alpha-1 antitrypsin deficiency (AATD) in a late-breaking oral session at the European Respiratory Society (ERS) Congress 2026, held September 5–9, 2026 in Barcelona, Spain. BEAM-302 is described by Beam as its lead genetic disease program, a liver-targeting base-editing therapy intended to address both lung and liver manifestations of AATD by restoring AAT levels and function while eliminating toxic Z-AAT.
The late-breaking presentation, titled “First-in-Human in vivo Gene Editing for Severe Alpha-1 Antitrypsin Deficiency (AATD): Initial Data from the Phase 1/2 Study of BEAM-302”, is scheduled in the session “Understanding Treatment Response in Airway Diseases: from Mechanisms to Clinical Biomarkers” on Tuesday, September 8, 2026, from 9:30–10:45 a.m. CEST, and will be delivered by John Hurst, M.D., Ph.D., University College London. Beam also plans an investor conference call and webcast on Tuesday, September 8, 2026, at 7:00 a.m. ET, accessible via the Events section of its website, with a replay available afterward.
Beam Therapeutics (Nasdaq: BEAM) received U.S. FDA clearance of its IND application for BEAM-304, an in vivo base-editing candidate for treating phenylketonuria (PKU).
BEAM-304 uses lipid nanoparticle delivery to correct PAH gene mutations, with preclinical mouse data showing normalization of plasma phenylalanine at clinically relevant doses. A Phase 1/2 trial in PKU patients is planned.
Beam Therapeutics (Nasdaq: BEAM) presented updated Phase 1/2 data for its base-editing candidate BEAM-302 in alpha-1 antitrypsin deficiency (AATD) at the ATS 2026 conference.
The update covers single-dose cohort safety, durability of efficacy and reduced human neutrophil elastase activity, and outlines plans for an FDA accelerated approval path with a pivotal expansion starting in 2H 2026.
Beam Therapeutics (NASDAQ: BEAM) became an inaugural industry sponsor of AlphaDetect, a nonprofit powered by the Alpha-1 Foundation. The sponsorship supports scaling routine, targeted detection of Alpha-1 Antitrypsin Deficiency (Alpha-1) in liver and lung disease patients through free genetic testing, provider support, and enhanced detection protocols aligned with clinical guidelines.
Beam Therapeutics (Nasdaq: BEAM) announced that CEO John Evans will present at the 2026 RBC Capital Markets Global Healthcare Conference on May 20, 2026, at 10:30 a.m. ET in New York.
A live webcast will stream via the investor section of www.beamtx.com and remain archived for 60 days.
Beam Therapeutics (Nasdaq: BEAM) will present updated biomarker data from the Phase 1/2 BEACON trial of ristoglogene autogetemcel (risto-cel) in sickle cell disease at EHA2026, June 11-14, 2026, in Stockholm.
Presentations highlight red blood cell health, sickling and rheology parameters, and fetal hemoglobin correlations.
Beam Therapeutics (Nasdaq: BEAM) reported Q1 2026 results and program updates on May 7, 2026. Key items: BEAM-302 selected 60 mg as the optimal biological dose with a pivotal cohort planned in H2 2026; BEACON risto-cel data published in NEJM and a BLA is expected as early as year-end 2026. Cash, cash equivalents and marketable securities were $1.2 billion, with runway expected into mid-2029.
Beam Therapeutics (Nasdaq: BEAM) announced NEJM publication of interim Phase 1/2 BEACON data for risto-cel in sickle cell disease on April 1, 2026. Data from 31 patients (follow-up 0.3–20.4 months) show mean HbF >60%, mean HbS 40%, no investigator-reported severe VOCs post-engraftment, median 1 cell collection, median 2.9 months to product release, median 4.5 months to dosing. Beam expects a U.S. BLA submission as early as year-end 2026; risto-cel holds orphan drug and RMAT designations and is in the FDA CDRP program.
Beam Therapeutics (Nasdaq: BEAM) reported updated Phase 1/2 data for BEAM-302 in alpha-1 antitrypsin deficiency showing a well-tolerated safety profile and robust biomarker responses supporting pivotal advancement.
Key findings: 60 mg produced mean steady-state total AAT of 16.1 µM, >94% M-AAT composition, ~84% reduction in Z-AAT, and all patients above the 11 µM protective threshold; pivotal cohort expected H2 2026.