ASGCT 2021 | Vector-based therapies for metabolic disorders

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.