Beam Therapeutics Announces Positive Topline Pivotal Data from BEACON Phase 1/2 Trial of Risto-cel

Beam Therapeutics reported positive topline results from the BEACON Phase 1/2 trial of risto-cel, its investigational one-time autologous cell therapy for sickle cell disease, saying the study met its primary efficacy endpoint. The company plans to submit a Biologics License Application to the FDA as early as the end of 2026; if filed, it could be the first BLA for a base-editing therapy. Detailed pivotal results are expected at a medical meeting in 2027.