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ASH 2022 | Phase I study of lentiviral-mediated gene therapy in patients with severe pyruvate kinase deficiency

Ami Shah, MD, Lucile Packard Children’s Hospital, Palo Alto, CA, summarizes the findings of a Phase I study assessing the efficacy of lentiviral-mediated gene therapy with RP-L301 in adults and children with pyruvate kinase deficiency who have undergone splenectomy and require blood transfusions (NCT04105166). Overall, the study showed a marked improvement in hemoglobin levels and quality of life (QoL), with no serious adverse events and no requirements for red blood cell transfusions post-engraftment. This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

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Advisory board for Bluebird Bio and Vertex Pharmaceuticals.