Facing potential chemistry, manufacturing, & control (CMC) development challenges with recombinant adeno-associated viral vectors: available regulatory guidance & recommendations

These hypothetical ‘case studies’ and topics illustrate some of the many challenges faced by developers of recombinant adeno-associated virus (rAAV)-based vectors in recent years. Direction to available guidance and resources are provided within this article, along with possible approaches towards mitigating such regulatory risks throughout AAV product development. Whilst the generalized issue topics are not specific to any particular investigator or sponsor, it is intended that they be topical and relevant to challenges that could be faced by developers, including those from smaller enterprises. The breadth of the case studies (or challenges faced by developers) is not exhaustive, and per issue, the approaches below should be modified and developed upon per investigational AAV-based gene therapy medicinal product (GTMP) along with recommendation to seek early endorsement from appropriate health authorities. Most of the references to regional health authority regulatory guidelines are focused to the US Food and Drug Administration (FDA) and European Medicines Agency (EMA). 

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