Prestigious Publication Reinforces risto-cel's Durable, Differentiated Clinical Data for the Treatment of SCD with Severe Vaso-occlusive Crises (VOCs)

Initially Presented at ASH, Data from 31 Patients with SCD Demonstrated Deep Resolution of Red Blood Cell Dysfunction and Reduced Time in Hospital Driven by a Median of One Cell Collection Cycle and Rapid Engraftment

U.S. Biologics License Application (BLA) Submission for risto-cel Expected as Early as Year-End 2026

CAMBRIDGE, Mass., April 01, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the publication of data from the ongoing Phase 1/2 BEACON clinical trial evaluating ristoglogene autogetemcel (risto-cel, formerly known as BEAM-101) for the treatment of sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs) in The New England Journal of Medicine (NEJM). Risto-cel is an investigational autologous cell therapy with a potential best-in-class profile for the treatment of SCD.

"The publication of these interim data from the BEACON trial in The New England Journal of Medicine underscores the potential of risto-cel to make a transformational difference in the lives of patients living with SCD by reducing severe pain crises and the progressive complications that impact quality of life and lifespan," said Matthew M. Heeney, M.D., the associate chief of hematology at the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and corresponding author of the NEJM publication. "In the data presented, risto-cel demonstrated an acceptable safety profile consistent with myeloablative conditioning, as well as very encouraging efficacy with rapid engraftment and induction of non-sickling fetal hemoglobin to levels sustained above 60% with concomitant reduction of sickle hemoglobin to below 40%, achieving a protective hemoglobin ratio similar to sickle cell trait. These changes resulted in improved hemolysis parameters, resolution of anemia, and no reported severe VOCs following engraftment. Risto-cel has the potential to meaningfully alter the pathophysiology of the disease and improve outcomes for patients with SCD."