Quality, non-clinical and clinical issues relating specifically to recombinant adeno-associated viral vectors - Scientific guideline

This document aims to discuss quality, non-clinical and clinical issues that should be considered during the development of medicinal products derived from adeno-associated viral vectors. It indicates requirements that might be expected at the time of a marketing authorisation application.

Keywords: Adeno-associated virus, self complementary adeno-associated virus, recombinant adeno-associated virus, production systems, quality, non-clinical, clinical, follow-up, tissue tropism, germ-line transmission, environmental risk, immunogenicity, biodistribution, shedding, animal models, persistence, reactivation, advanced therapy medicinal products (ATMPs), gene therapy medicinal products