Beam reports positive Phase 1/2 results for base-edited sickle cell therapy

Beam Therapeutics announced positive topline results on Nov. 10, 2026, from its BEACON Phase 1/2 study of risto-cel (formerly BEAM-101), an investigational, one-time base-edited cell therapy for severe sickle cell disease.

The trial met its primary efficacy endpoint:
freedom from severe vaso-occlusive crises (pain crises) for at least 12 consecutive months. It also met reported secondary efficacy endpoints, including freedom from inpatient hospitalization for severe crises, and all primary safety endpoints at the interim analysis.

By the Oct. 20 data cutoff, 49 patients—39 adults and 10 adolescents—had been treated; 31 had completed the follow-up needed for the primary endpoint. Beam has not disclosed what proportion of those 31 met it, so the topline announcement does not show the size of the response in detail.

Risto-cel uses patients’ own blood-forming stem cells, edited outside the body to increase protective fetal hemoglobin. Treatment involves stem-cell transplant procedures and conditioning chemotherapy, so the positive result does not mean the therapy is risk-free or already approved.

Beam said it is preparing a U.S. biologics license application, potentially by the end of 2026. The therapy remains investigational; detailed results are expected at a future medical meeting.