WASHINGTON (dpa-AFX) - Beam Therapeutics Inc. (BEAM) announced new safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs).
Updated data from seven patients treated with investigational base-editing therapy 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. No vaso-occlusive crises were reported post-engraftment.
The company believes the early data for BEAM-101 are a testament to the potential of our base-editing technology to provide a differentiated option for sickle cell patients, having demonstrated a robust increase in fetal hemoglobin of greater than 60%, a decrease in hemoglobin S to less than 40% and resolution of anemia in all patients. Additionally, the data from ESCAPE nongenotoxic conditioning program highlight commitment to expanding access to treatment by decreasing the burden and complications patients potentially face when undergoing transplantation.
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