Vertex Pharmaceuticals (VRTX) announced longer-term data for CASGEVY from global clinical trials in people with severe sickle cell disease or transfusion-dependent beta thalassemia. CASGEVY is the first and only approved CRISPR/Cas9 gene-edited therapy. The results, presented at the American Society of Hematology Annual Meeting and Exposition, continue to demonstrate the transformative, durable clinical benefits of CASGEVY. The longest follow up for both SCD and TDT patients now extends more than 5 years, with a median of 33.2 months and 38.1 months, respectively. In SCD, 39/42 evaluable patients were free from vaso-occlusive crises for at least 12 consecutive months in CLIMB-121 and CLIMB-131 combined. The mean duration of VOC-free was 30.9 months, with a maximum of 59.6 months. The three evaluable patients who have not achieved VF12 have derived meaningful clinical benefit including by reducing their rate of hospitalization for VOCs by 91%, 71% and 100%. In TDT, 53/54 evaluable patients achieved transfusion-independence for at least 12 consecutive months with a weighted average hemoglobin of at least 9 g/dL in CLIMB-111 and CLIMB-131 combined. The mean duration of transfusion independence was 34.5 months, with a maximum of 64.1 months. The one evaluable patient who has not yet achieved TI12 has been transfusion free for 8.2 months. Both SCD and TDT patients reported sustained and clinically meaningful improvements in their quality of life, including physical, emotional, social/family and functional well-being, and overall health status. The safety profile of CASGEVY continues to be generally consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. Patients continue to demonstrate stable levels of fetal hemoglobin and allelic editing across all ages and genotypes in the trials.
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