Beam Therapeutics announces new data from BEACON Phase 1/2 trial

Beam Therapeutics (BEAM) announces new safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 in patients with sickle cell disease with severe vaso-occlusive crises. The data will be shared in a poster presentation today at the European Hematology Association 2025 Congress in Milan. New data from the BEACON trial with more patients and longer follow-up provide further demonstration of the strong clinical profile for the investigational base-editing therapy BEAM-101, as initially established in previously announced data at the 66th American Society of Hematology Annual Meeting and Exposition in December 2024. Updated data from 17 patients treated with BEAM-101 demonstrated robust and durable increases in fetal hemoglobin and reductions in sickle hemoglobin, rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis and oxygen delivery. Patients required a median of one mobilization cycle. No VOCs were reported post-engraftment. BEAM-101 is manufactured using an advanced, largely automated process that has demonstrated consistently high yields and viability, enabling successful manufacturing for all patients in the BEACON clinical trial as of the data cut-off. A summary of the results from the ongoing clinical study is provided below.