Abstract
Adeno-associated virus (AAV)-based gene therapy (GT) products can effectively deliver transgenes to replace lost or defective genes, providing hope for patients. However, there is limited research on analytical methods for pre-clinical and clinical studies which could impede their development success. During the development of AAV-based GT products, it is crucial to define the biodistribution and shedding profiles following treatment. In this review, we summarize PK studies for the AAV-based GT products approved so far by U.S. Food and Drug Administration and European Medicines Agency. We then comparatively review the analytical methods employed to evaluate AAV vectors’ biodistribution and shedding of approved GT products. We further make recommendations on choice of methodologies for future GT product development based on the study data and the currently available regulatory guidance.
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