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12 BioPharm International eBook October 2020 www.biopharminternational.com Viral Vector API Characterization of Product-Related Impurities FDA's final CMC guidance sets expectations for manufacturing and quality for human gene therapy INDs. This article reviews existing analytical applications, focusing on viral vector characterization of impurities. I n January 2020, the FDA issued Final Guidance for Industry: Chemistry Manufacturing and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) and other guidance documents for human gene therapies, which set expectations for manufacturing and demonstration of quality (1). These products are applied to deliver genetic material (transgenes) or modified cells that have been designed to alleviate or even eliminate a broad range of diseases that are rooted in the genetics of individuals or populations of patients. This article focuses on one of these guidance docu- ments, reviewing aspects of the chemistry, manufactur- ing and control (CMC) final guidance (2). The authors' goal is to assess existing and evolving analytical applica- tions and technologies and their associated challenges towards ensuring that viral vectored gene therapy drug substances are well characterized for purity and potency (as outlined in Section V.A.3.b.ii of FDA's CMC guidance). The analytical methods discussed here are used to identify and quantify the potency and product-related impurities within recombinant adeno-associated virus (AAV) vector gene therapy products, and the potential PETER WUNDERLI, JUN LU, JIE DING, MERCEDES AMES, MARC WOLMAN, JARED FINGER, AND STEPHEN GACHERU Regulatory Sourcebook Regulatory Guidance PETER WUNDERLI is research fellow; JUN LU is director; JIE DING is associate director; MERCEDES AMES and MARC WOLMAN are senior research scientists; JARED FINGER is principal scientist, and STEPHEN GACHERU,* stephen.gacheru@ ppd.com, 608.203.3131, is vice president, all with PPD Laboratories' GMP Lab, Middleton, Wisconsin. *To whom all correspondence should be addressed. luchschenF - Stock.adobe.com