The FDA Has Granted Orphan Drug Designation To Beam Therapeutics' Beam-101, An Investigational Genetically Modified Cell Therapy For Sickle Cell Disease

Data from seven patients treated with BEAM-101 in the BEACON clinical trial were presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2024. Updated clinical data have been accepted for presentation at the European Hematology Association (EHA) 2025 Congress in June. Treatment with BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis. The initial safety profile of BEAM-101 was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation. Manufactured in Beam's North Carolina facility, BEAM-101 uses an advanced, largely automated process that has demonstrated consistently high yields and viability, enabling successful BEAM-101 manufacturing for patients in the BEACON clinical trial. Beam expects to dose 30 patients in the ongoing BEACON trial by mid-2025.