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rAAV Vector Product Characterization and Stability Studies

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Adeno-Associated Virus

Part of the book series: Methods in Molecular Biology ((MIMB,volume 807))

Abstract

Recombinant adeno-associated viral (rAAV) vectors mediate the safe and long-term correction of genetic diseases following a single administration. Preclinical studies in animal models and human trials have shown rAAV vector persistence and safety. In some trials, sustained or transient transgene expression has been demonstrated in humans treated for alpha-1 antitrypsin deficiency, LPL deficiency, hemophilia B and cystic fibrosis, and sustained correction of inherited blindness has been reported by three groups. For human use, rAAV vectors are manufactured and tested in compliance with current Good Manufacturing Practices as outlined in the Code of Federal Regulations (21CFR) or European Good Manufacturing Practices (Eudralex, Volume 4, GMP Guidelines, 2003/94/CE and 91/356/EEC). Manufacturing control, as well as product quality is evaluated by quality control testing and all manufacturing, facilities, and testing activities are reviewed by the quality assurance department. In-process specifications are set and in-process testing is conducted to confirm that the manufacturing process is controlled, aseptic, and performs consistently. Final product is tested to ensure release specifications are met for identity, safety, purity, potency, and stability.

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Acknowledgments

We acknowledge Mahuya Bose and Weiyi Ni for insightful discussions. ROS is an inventor on patents related to recombinant AAV technology and owns equity in a gene therapy company that is commercializing AAV for gene therapy applications. To the extent that the work in this manuscript increases the value of these commercial holdings ROS has a conflict of interest.

Author information

Authors and Affiliations

  1. Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL, USA

    Richard O. Snyder

  2. GenoSafe SAS, EVRY Cedex, France

    Muriel Audit

  3. Center of Excellence for Regenerative Health Biotechnology, University of Florida, Alachua, FL, USA

    Richard O. Snyder & Joyce D. Francis

Authors
  1. Richard O. Snyder
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  2. Muriel Audit
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  3. Joyce D. Francis
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Corresponding author

Correspondence to Richard O. Snyder .

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Editors and Affiliations

  1. Center of Excellence for Regenerative, Health Biotechnology (CERHB), University of Florida, Innovation Drive 13706, Alachua, 32615, Florida, USA

    Richard O. Snyder

  2. INSERM U649, Labo. Thérapie Génique, CHU Hôtel Dieu, bd. Jean Monnet 30, Nantes CX 1, 44035, France

    Philippe Moullier

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Snyder, R.O., Audit, M., Francis, J.D. (2012). rAAV Vector Product Characterization and Stability Studies. In: Snyder, R., Moullier, P. (eds) Adeno-Associated Virus. Methods in Molecular Biology, vol 807. Humana Press. https://doi.org/10.1007/978-1-61779-370-7_17

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  • DOI: https://doi.org/10.1007/978-1-61779-370-7_17

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  • Publisher Name: Humana Press

  • Print ISBN: 978-1-61779-369-1

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