Beam Therapeutics Inc. has announced new safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101, an investigational base-editing therapy for sickle cell disease $(SCD)$ with severe vaso-occlusive crises (VOCs). The updated data, which will be presented in a poster session at the European Hematology Association 2025 Congress in Milan, involves 17 patients. All patients treated with BEAM-101 demonstrated induction of fetal hemoglobin (HbF) levels above 60%, a reduction in sickle hemoglobin (HbS) to below 40%, and resolution of anemia. The company reports that patients experienced rapid neutrophil and platelet engraftment and no VOCs post-engraftment. Enrollment is now complete in both adult and adolescent cohorts, with 30 patients expected to be dosed by mid-2025. Beam will host an investor webcast on June 13, 2025, to discuss these findings.
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