Gloria Gonzalez-Aseguinolaza – VJRegenMed

Enhancing AAV gene therapy in children with inborn errors of metabolism

admin_vjr_user — Wed, 18 Aug 2021 16:32:47 +0000

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.

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

admin_vjr_user — Wed, 18 Aug 2021 16:32:46 +0000

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.

Vector-based therapies for metabolic disorders

admin_vjr_user — Mon, 16 Aug 2021 08:50:47 +0000

Gloria Gonzalez-Aseguinolaza, PhD, Universidad de Navarra, Pamplona, Spain & Vivet Therapeutics, Paris, France, explains that the delivery of non-viral vectors containing small interfering RNAs (siRNAs), which inhibit toxic metabolite production, are an effective treatment option for inborn errors of metabolism such as acute intermittent porphyria. Alternative strategies include viral vector mediated gene therapy using adeno-associated vectors (AAVs), however vector transduction efficiency and gene integration must be improved to effectively treat inborn errors of metabolism. Other challenges associated with AAVs include inflammatory responses to high vector doses and exclusion of participants with anti-AAV antibodies. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.