Gene therapy a potential 'functional cure' for sickle cell d

Treatment with exagamglogene autotemcel conferred clinically meaningful increases in fetal hemoglobin and total hemoglobin levels among patients with beta-thalassemia and sickle cell disease, results of two pivotal studies showed. The latest follow-up data from the CLIMB THAL-111 and CLIMB-SCD-121 trials — presented at American Society for Gene & Cell Therapy Annual Meeting —

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