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Abstract
Gene therapy, involving the transfer of genetic materials into a patient to alter gene or protein expression, is a promising approach for treating a variety of human diseases. In vivo gene therapy uses gene-based materials to modify target cell genomes. Adeno-Associated Virus (AAV) vectors, known as the safest and the most effective vector to deliver genes of interest into a broad range of cell types, are gaining prominence. Luxturna's the U.S. Food and Drug Administration (FDA) approval marked a turning point for AAV-based gene therapy. Subsequently, Zolgensma and Hemgenix received approval from both the FDA and the European Medicines Agency (EMA), increasing interest in AAV based gene therapies for rare or untreatable diseases. Despite this progress, the regulatory and clinical trial processes for AAV-based gene therapy drugs are underexplored. Our study compares the approval processes of three prominent AAVbased gene therapy drugs: Luxturna, Zolgensma, and Hemgenix. We found that all three received expedited approvals from the FDA and EMA, primarily based on meticulously designed clinical trials demonstrating safety and efficacy. These trials underwent multiple adaptations to meet tight timelines, deviating from traditional regulatory pathways. These findings offer valuable insights for domestic AAV-based gene therapy developers, helping them refine clinical strategies for future innovations. This knowledge would further guide the development and regulation of future AAV-based gene therapies.
Published Version
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