Beam Therapeutics(US:BEAM) Globenewswireยท2025-06-13 11:00Core Insights - Beam Therapeutics Inc. announced new safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 for treating sickle cell disease (SCD), demonstrating strong clinical outcomes and a consistent safety profile [2][3][4] Efficacy and Safety Data - All patients treated with BEAM-101 achieved hemoglobin F (HbF) induction levels exceeding 60% and a reduction in sickle hemoglobin (HbS) to below 40%, with no vaso-occlusive crises reported post-engraftment [3][5][11] - Patients required a median of one mobilization cycle for treatment, with rapid neutrophil and platelet engraftment observed, taking a median of 16.5 days and 19.5 days respectively [11] - Key markers of hemolysis normalized or improved in all patients, indicating significant enhancement in oxygen delivery to tissues [11] Clinical Trial Progress - Enrollment in both adult and adolescent cohorts of the BEACON trial is complete, with 26 patients dosed as of June 13, 2025, and an expectation to dose 30 patients by mid-2025 [7][8] - The follow-up period for treated patients ranged from 0.2 to 15.1 months, with durable responses observed in HbF and HbS levels [4][11] Manufacturing and Process - BEAM-101 is manufactured using an advanced, largely automated process that has shown high yields and viability, facilitating successful manufacturing for all patients in the trial [3][11] - The safety profile of BEAM-101 aligns with that of busulfan conditioning and autologous hematopoietic stem cell transplantation, with common treatment-emergent adverse events being consistent with busulfan [11] Company Vision and Future Plans - Beam Therapeutics aims to deliver life-long cures through its precision genetic medicines platform, focusing on base editing technology [12] - The company is committed to advancing its BEAM-101 program and plans to share additional data from the trial by the end of 2025 [4][7]