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ASGCT 2021 | Lentiviral gene therapy for pyruvate kinase deficiency

Jose-Carlos Segovia, PhD, CIEMAT, CIBERER & IIS- FJD, Madrid, Spain, describes ongoing research into the use of gene therapy technologies for pyruvate kinase deficiency (PKD), a rare inherited hemolytic anemia. He describes an ongoing global Phase I clinical trial (NCT04105166) of RP-L301, an investigational ex vivo lentiviral gene therapy, in which two patients with PKD have been treated thus far. Preliminary data obtained from Patient 1 and 2 at 6- and 3-months follow up, respectively, demonstrated the normalization of erythrocyte counts and hemoglobin levels, and that the treatment was well tolerated. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.


Jose-Carlos Segovia is a consultant for and has received research funding from Rocket Pharmaceuticals, Cranbury, NJ.