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ASGCT 2021 | Enhancing AAV gene therapy in children with inborn errors of metabolism

Gloria Gonzalez-Aseguinolaza, PhD, Universidad de Navarra, Pamplona, Spain & Vivet Therapeutics, Paris, France, explains that a key challenge associated with treating inborn errors of metabolism in children using non-integrating vector-based gene therapy is liver growth and therefore hepatocyte addition, which results in the dilution of therapeutic effect and need for re-administration. Subsequent doses are, however, typically less effective due to the presence of neutralizing antibodies following the first injection. Approaches being investigated to overcome this challenge include the use of ImmTOR tolerogenic nanoparticles carrying rapamycin which inhibit neutralising antibody formation, as well as strategies to reduce adeno-associated vector (AAV) genome loss during liver growth. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.


Gloria Gonzalez-Aseguinolaza is the cofounder and CSO of Vivet Therapeutics and holds shares of the company.