Biomedicines 2014, 2(1), 80-97; doi:10.3390/biomedicines2010080
Review

Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment

Received: 13 December 2013; in revised form: 20 February 2014 / Accepted: 20 February 2014 / Published: 3 March 2014
(This article belongs to the Special Issue Feature Papers)
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Abstract: Adeno-associated virus (AAV)-based vectors expressing therapeutic genes continue to demonstrate great promise for the treatment of a wide variety of diseases and together with other gene transfer vectors represent an emerging new therapeutic paradigm comparable in potential impact on human health to that achieved by recombinant proteins and vaccines. A challenge for the current pipeline of AAV-based investigational products as they advance through clinical development is the identification, characterization and lot-to-lot control of the process- and product-related impurities present in even highly purified preparations. Especially challenging are AAV vector product-related impurities that closely resemble the vector itself and are, in some cases, without clear precedent in established biotherapeutic products. The determination of acceptable levels of these impurities in vectors prepared for human clinical product development, with the goal of new product licensure, requires careful risk and feasibility assessment. This review focuses primarily on the AAV product-related impurities that have been described in vectors prepared for clinical development.
Keywords: AAV vectors; impurities; clinical trials
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MDPI and ACS Style

Wright, J.F. Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment. Biomedicines 2014, 2, 80-97.

AMA Style

Wright JF. Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment. Biomedicines. 2014; 2(1):80-97.

Chicago/Turabian Style

Wright, J. F. 2014. "Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment." Biomedicines 2, no. 1: 80-97.

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