The number of gene therapy (GTx) modality therapies in development has grown significantly in the last few years. Adeno-associated virus (AAV)-based delivery approach has become most prevalent among other virus-based GTx vectors. Several regulatory guidelines provide the industry with general considerations related to AAV GTx development including discussion and recommendations related to highly diverse bioanalytical support of the AAV-based therapeutics. This includes assessment of pre- and post-treatment immunity, evaluation of post-treatment viral shedding and infectivity, as well as detection of transgene protein expression. An overview of the current regulatory recommendations as found in currently active and published draft US FDA and EMA guidance or guideline documents is presented herein.

Keywords:

Papers of special note have been highlighted as: •• of considerable interest

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Received 31 May 2019

Accepted 6 September 2019

Published online 25 October 2019

Published in print November 2019

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Financial & competing interests disclosure

All the authors are employees of Pfizer, Inc., and may own respective stock or other form of equity. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

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Bioanalysis of adeno-associated virus gene therapy therapeutics: regulatory expectations

Abstract

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