Beam Therapeutics to Present Updated Data from BEACON Phase 1/2 Trial of BEAM-101 in Sickle Cell Disease at American Society of Hematology (ASH) Annual Meeting

Core Insights - Beam Therapeutics Inc. is set to present updated safety and efficacy data for BEAM-101, a gene therapy for sickle cell disease (SCD), at the 67th ASH Annual Meeting in December 2025 [1][2] - BEAM-101 aims to provide a one-time, disease-modifying treatment for patients suffering from severe vaso-occlusive crises associated with SCD [2][3] Company Overview - Beam Therapeutics is a biotechnology company focused on precision genetic medicines, utilizing a proprietary base editing technology to develop therapies [5][6] - The company has established a fully integrated platform for gene editing, delivery, and manufacturing capabilities [5] Product Details - BEAM-101 is an investigational therapy that modifies autologous CD34+ hematopoietic stem and progenitor cells to increase fetal hemoglobin production, potentially alleviating symptoms of SCD [3] - The ongoing BEACON Phase 1/2 study is evaluating the safety and efficacy of BEAM-101 in adult patients with severe SCD [3] Disease Context - Sickle cell disease affects approximately eight million people globally and is characterized by severe complications including anemia, pain crises, and organ failure [2][4] - In the U.S., SCD is the most common inherited blood disorder, impacting an estimated 100,000 individuals [4]