Lentiviral gene therapy for pyruvate kinase deficiency

admin_vjr_user — Fri, 14 May 2021 15:12:26 +0000

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.

A novel lentiviral therapeutic approach to primary hyperoxaluria type 1

admin_vjr_user — Fri, 14 May 2021 14:11:24 +0000

Jose-Carlos Segovia, PhD, CIEMAT, CIBERER & IIS- FJD, Madrid, Spain, discusses the limitations associated with the use of adeno-associated virus vectors when targeting primary hyperoxaluria type 1 (PH1), a rare genetic disorder of the liver caused by mutations in the AGXT gene. Given the high integrating capacity of lentiviral vectors, they are being investigated in a preclinical mouse model as a novel therapeutic approach for PH1. Preliminary studies have demonstrated that treatment with hepatocyte-specific lentiviral gene therapy resulted in high levels of gene correction, although further work is required to improve transduction efficiency and enhance therapeutic benefit. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Jose-Carlos Segovia – VJRegenMed

Lentiviral gene therapy for pyruvate kinase deficiency

A novel lentiviral therapeutic approach to primary hyperoxaluria type 1