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An Overview of rAAV Vector Product Development for Gene Therapy

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Regenerative Medicine for Degenerative Muscle Diseases

Part of the book series: Stem Cell Biology and Regenerative Medicine ((STEMCELL))

Abstract

The drug product development pathway involves proof-of-concept efficacy studies, toxicology studies, and human clinical trials conducted in preparation for commercialization. For time and financial efficiency, a perspective of clinical use, product licensure, and commercialization is beneficial at the outset. For gene transfer products, an understanding of the unmet need for the disease target, the market and competing products, the vector configuration, patient population profile, organ target, delivery method, and time and resources involved is essential. Addressing these variables leads to interconnected strategies and plans for business and finance, regulatory affairs, product supply, R&D, clinical investigations, and commercial launch. Establishing an organization with experienced personnel who have defined responsibilities, departments that are working together but are independently responsible, a network of consultants and contract organizations, and a high level of communication is essential to ensure the quality of the product and protection of patients and give the highest chance for success.

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Conflict of Interest Statement

RS is an inventor on patents related to recombinant AAV technology. RS 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, RS has a conflict of interest.

Author information

Authors and Affiliations

  1. Department of Molecular Genetics and Microbiology, University of Florida, College of Medicine, 1600 SW Archer Road, Gainesville, FL, 32610-0266, USA

    Richard O. Snyder Ph.D.

  2. Atlantic Gene Therapies, INSERM UMR 1089, Université de Nantes, CHU de Nantes, Nantes, 44007, France

    Richard O. Snyder Ph.D.

  3. Center of Excellence for Regenerative Health Biotechnology, University of Florida, Alachua, FL, 32615, USA

    Richard O. Snyder Ph.D.

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  1. Richard O. Snyder Ph.D.
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Correspondence to Richard O. Snyder Ph.D. .

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Editors and Affiliations

  1. Department of Rehabilitation Medicine, University of Washington Department of Rehabilitation Medicine, WINSTON SALEM, North Carolina, USA

    Martin K. Childers

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Snyder, R.O. (2016). An Overview of rAAV Vector Product Development for Gene Therapy. In: Childers, M. (eds) Regenerative Medicine for Degenerative Muscle Diseases. Stem Cell Biology and Regenerative Medicine. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-3228-3_2

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