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Review

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

1
Center for Cellular and Molecular Therapeutics, the Children's Hospital of Philadelphia, ARC1216C, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA
2
Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, ARC1216C, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA 
Biomedicines 2014, 2(1), 80-97; https://doi.org/10.3390/biomedicines2010080
Received: 13 December 2013 / Revised: 20 February 2014 / Accepted: 20 February 2014 / Published: 3 March 2014
(This article belongs to the Special Issue Feature Papers)
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. View Full-Text
Keywords: AAV vectors; impurities; clinical trials 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. https://doi.org/10.3390/biomedicines2010080

AMA Style

Wright JF. Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment. Biomedicines. 2014; 2(1):80-97. https://doi.org/10.3390/biomedicines2010080

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. https://doi.org/10.3390/biomedicines2010080

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