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초록

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

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