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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) 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.
Beam Therapeutics (Nasdaq: BEAM) reported Q4 and full-year 2025 results, noted a strengthened balance sheet with $1.25B in cash and marketable securities, and announced a new liver-targeted program BEAM-304 for phenylketonuria (PKU) with an IND expected in 2026.
Beam secured a $500M senior secured facility with Sixth Street, expects a minimum $200M draw, and reiterated plans for a potential BLA submission for risto-cel as early as year-end 2026 and BEAM-302 pivotal next steps in Q1 2026.
Beam Therapeutics (Nasdaq: BEAM) secured a $500 million senior secured credit facility with Sixth Street to support the anticipated commercial launch of ristoglogene autogetemcel (risto-cel) in sickle cell disease. $100 million funded at close; up to $300 million tied to clinical, regulatory, and commercial milestones; plus a $100 million option. The seven-year facility matures in early 2033, bears ~10% annual interest, requires no scheduled amortization, is secured by first‑priority liens on substantially all assets, and includes mandatory prepayments from certain extraordinary proceeds.
Beam Therapeutics (Nasdaq: BEAM) will host an investor webcast on Tuesday, February 24, 2026 at 8:00 a.m. ET to announce its fourth quarter and full year 2025 financial results and a new liver-targeted genetic disease program.
The live webcast will be available in the investor section of the company's website and will be archived for 60 days after the presentation.