Beam Therapeutics' risto-cel meets primary endpoints in sickle cell trial
BEAM•Beam Therapeutics said its BEACON Phase 1/2 trial of risto-cel met its primary efficacy endpoint and all primary safety and secondary endpoints. The company plans to submit a U.S. biologics license application as early as year-end 2026.
1. Trial results
Beam Therapeutics reported positive pivotal results from its BEACON Phase 1/2 trial of risto-cel, an investigational one-time treatment for sickle cell disease. The trial met its primary goal of sustained freedom from severe vaso-occlusive crises, as well as all primary safety and secondary endpoints; detailed pivotal results are planned for a medical meeting in 2027.
