Beam Therapeutics’ risto-cel shows efficacy in Sickle Cell Disease trial

Beam Therapeutics (BEAM) announced the publication of data from the ongoing Phase 1/2 BEACON clinical trial evaluating ristoglogene autogetemcel – risto-cel, formerly known as BEAM-101 – for the treatment of sickle cell disease, or SCD with severe vaso-occlusive crises, or VOCs, in The New England Journal of Medicine. Risto-cel is an investigational autologous cell therapy with a potential best-in-class profile for the treatment of SCD. In the data presented, risto-cel demonstrated an acceptable safety profile consistent with myeloablative conditioning, as well as “very encouraging” efficacy. These changes resulted in improved hemolysis parameters, resolution of anemia, and no reported severe VOCs following engraftment. Patients achieved “rapid and robust” bone marrow reconstitution post-risto-cel treatment, and no patients experienced any investigator-reported severe VOCs post-engraftment. Patients achieved mean hemoglobin F levels above 60% and a mean durable reduction in corresponding hemoglobin S below 40%. Key markers of hemolysis normalized or improved in all patients following risto-cel treatment. Sickling parameters all decreased in the blood following risto-cel treatment to levels comparable to those seen in individuals with sickle cell trait. The safety profile of risto-cel was consistent with busulfan conditioning, autologous hematopoietic stem cell transplantation and underlying SCD.