LentiGlobin gene therapy for transfusion-dependent β-Thalassemia: Update from the northstar HGB-204 phase 1/2 clinical study

      Background: Gene therapy could provide a transformative treatment for patients with transfusion-dependent β-thalassemia (TDT). HGB-204 is a multi-center Phase 1/2 clinical study investigating the safety and efficacy of LentiGlobin BB305 Drug Product (DP) in patients with TDT. We report 3.7–27.0 months follow up on the study's full cohort.
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