ASGCT 2021 | Combining rAAV gene therapy with standard-of-care for the treatment of citrullinemia

Gloria Gonzalez-Aseguinolaza, PhD, Universidad de Navarra, Pamplona, Spain & Vivet Therapeutics, Paris, France, discusses ongoing research into gene therapies for citrullinemia type I (CTLN1) in pediatric patients, a rare autosomal recessive genetic disorder caused by mutations in the ASS1 gene, that can result in early-onset hyperammonemia crisis. Combining standard of care nitrogen scavenger therapy with arginine administration prior to administration of VTX-804 gene therapy, a recombinant adeno-associated virus (rAAV) vector expressing the human ASS1 enzyme, has been demonstrated to control initial disease symptoms and improve therapeutic efficacy in a mouse model. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.