Beam Therapeutics Reports Updated Data from BEACON Phase 1/2 Trial of ristoglogene autogetemcel (risto-cel) Highlighting Durable, Differentiated Profile in Sickle Cell Disease (SCD) at American Society of Hematology (ASH) Annual Meeting

Core Insights - Beam Therapeutics has announced updated safety and efficacy data for ristoglogene autogetemcel (risto-cel), a genetically modified cell therapy for sickle cell disease (SCD), showing significant clinical benefits and a durable response in patients [1][2] Efficacy and Safety Data - The BEACON Phase 1/2 trial included 31 patients with severe SCD, demonstrating a mean Hemoglobin F (HbF) induction of over 60% and a reduction in Hemoglobin S (HbS) to below 40% [1][3] - Patients experienced resolution of anemia, with total hemoglobin levels increasing rapidly and normalization of hemolysis markers [10] - No severe vaso-occlusive crises (VOCs) were reported post-engraftment, indicating a favorable safety profile [3][10] Treatment Process and Outcomes - Patients required a median of one stem cell collection cycle, with a median of three total collection days for manufacturing risto-cel, highlighting the efficiency of the process [3][5] - Rapid engraftment was observed, with a median time to neutrophil engraftment of 17.5 days and platelet engraftment at 19 days [3][10] - The treatment demonstrated durable, high editing efficiency, with mean peripheral blood editing rates of 67.4% at Month 6 and 72.8% at Month 12 [3][4] Company Overview - Beam Therapeutics focuses on precision genetic medicines through base editing technology, aiming to provide life-long cures for serious diseases [8] - The company is advancing a diversified portfolio of base editing programs, with risto-cel being a key investigational therapy for SCD [6][8]