Beam Therapeutics reported a positive late-stage readout for risto-cel, a base editing therapy for sickle cell disease, meeting its primary goal of “freedom from severe vaso-occlusive crises” with 12 months of follow-up in trial participants. Beam said secondary endpoints tied to reductions in hospital stays associated with VOCs also improved. The company plans to submit an approval application to the FDA by year-end. If approved, risto-cel would join a competitive gene- and cell-therapy landscape in sickle cell, where earlier entrants have faced adoption and real-world logistics challenges.