By Genoa Barrow | OBSERVER Senior Staff Writer - The FDA has approved the first two gene therapies to treat sickle cell disease, providing hope for patients, their families, and the entire sickle cell community, but the treatments are expensive and require specialized care to be effective.
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The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185). It was the second approval from FDA for a gene therapy for sickle cell disease.