Beam Therapeutics to Encore Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 in Sickle Cell Disease at 2025 Tandem Meetings of ASTCT and CIBMTR

Core Insights - Beam Therapeutics Inc. is advancing its investigational base-editing therapy BEAM-101 for sickle cell disease, with updated data to be presented at the 2025 Tandem Meetings [1][2] Company Overview - Beam Therapeutics is a biotechnology company focused on precision genetic medicines, utilizing a proprietary base editing technology that allows for precise genetic modifications without double-stranded breaks in DNA [5] - The company aims to establish a fully integrated platform for gene editing, delivery, and manufacturing capabilities [5] Product Details - BEAM-101 is a genetically modified cell therapy designed for severe sickle cell disease, utilizing autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited to increase fetal hemoglobin production [4] - The therapy aims to inhibit the transcriptional repressor BCL11A, leading to increased production of non-sickling fetal hemoglobin [4] Clinical Trial Information - The BEACON Phase 1/2 clinical trial is currently evaluating the safety and efficacy of BEAM-101, with previous data showing significant increases in fetal hemoglobin and improvements in hemolysis markers [2][4] - An oral presentation detailing the ongoing trial results will take place on February 12, 2025, by Dr. Ashish Gupta [3]