Beam Therapeutics (BEAM) announced new safety and efficacy data from its BEACON Phase 1/2 clinical trial of ristoglogene autogetemcel, formerly known as BEAM-101, an investigational genetically modified ex vivo base editing cell therapy, in patients with sickle cell disease, SCD, with severe vaso-occlusive crises, VOCs. The data will be shared today at the 67th American Society of Hematology, ASH, Annual Meeting and Exposition in Orlando. Key highlights: Updated Data from 31 Adult and Adolescent SCD Patients Treated with risto-cel Show Mean Hemoglobin F Induction of greater than60%, Hemoglobin S Reduction to less than40%, and Resolution of Anemia Durable for up to 20 Months; Patients Required a Median of One Cell Collection Cycle and Experienced Rapid Neutrophil and Platelet Engraftment; Safety Profile Remained Consistent with Busulfan Conditioning, Autologous Hematopoietic Stem Cell Transplantation and Underlying SCD
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