Beam Therapeutics reported positive topline results from its BEACON Phase 1/2 pivotal trial of risto-cel, an investigational one-time cell therapy for severe sickle cell disease (SCD). The trial met its primary efficacy endpoint: patients achieved at least 12 consecutive months without severe vaso-occlusive crises (VOCs).
The trial also met all primary safety endpoints. Risto-cel’s safety profile was consistent with previous findings.
Risto-cel uses base editing technology designed to increase production of non-sickling fetal hemoglobin. The results support the company’s plan to submit a Biologics License Application (BLA) to the FDA as early as the end of 2026.