Pharmaceutical Technology - October 2020

PharmTech - Regulatory Sourcebook - October

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52 Pharmaceutical Technology Regulatory Sourcebook October 2020 P h a r mTe c h . c o m Regulatory Guidance I n January 2020, FDA issued Final Guidance for Industry: Chem- istry 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 are 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 documents, review- ing aspects of the chemistry, manufacturing and control (CMC) final guidance (2). The authors' goal is to assess existing and evolving ana- lytical applications 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 quan- tify the potency and product-related impurities within recombinant adeno-associated virus (AAV) vector gene therapy products, and the potential impact of these impurities on product quality and performance. This article will highlight the characterization of: • Empty and partial viral particle contaminants relative to the quantity of full capsids • Quantitation of the total number of genomes present relative to the viral particles infectivity (i.e., those that are capable of delivering the gene of interest to cells). 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@; 608.203.3131, is vice-president, all with PPD Laboratories' GMP Lab, Middleton, WI. *To whom all correspondence should be addressed. LUCHSCHENF - STOCK.ADOBE.COM Viral Vector API Characterization of Product-Related Impurities Peter Wunderli, Jun Lu, Jie Ding, Mercedes Ames, Marc Wolman, Jared Finger, and Stephen Gacheru. 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.

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