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Gene Therapy

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New Bioprocessing Strategies: Development and Manufacturing of Recombinant Antibodies and Proteins

Part of the book series: Advances in Biochemical Engineering/Biotechnology ((ABE,volume 165))

Abstract

Gene therapy refers to a rapidly growing field of medicine in which genes are introduced into the body to treat or prevent diseases. Although a variety of methods can be used to deliver the genetic materials into the target cells and tissues, modified viral vectors represent one of the more common delivery routes because of its transduction efficiency for therapeutic genes. Since the introduction of gene therapy concept in the 1970s, the field has advanced considerably with notable clinical successes being demonstrated in many clinical indications in which no standard treatment options are currently available. It is anticipated that the clinical success the field observed in recent years can drive requirements for more scalable, robust, cost effective, and regulatory-compliant manufacturing processes. This review provides a brief overview of the current manufacturing technologies for viral vectors production, drawing attention to the common upstream and downstream production process platform that is applicable across various classes of viral vectors and their unique manufacturing challenges as compared to other biologics. In addition, a case study of an industry-scale cGMP production of an AAV-based gene therapy product performed at 2,000 L-scale is presented. The experience and lessons learned from this largest viral gene therapy vector production run conducted to date as discussed and highlighted in this review should contribute to future development of commercial viable scalable processes for vial gene therapies.

Barb Thorne and Ryan Takeya contributed equally to this work.

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Authors and Affiliations

  1. Thorne Bio-Consulting LLC, Sammamish, WA, USA

    Barb Thorne

  2. RKT Bioconsulting, LLC, Lynnwood, WA, USA

    Ryan Takeya

  3. Lonza Houston, Inc., Houston, TX, USA

    Francesca Vitelli & Xin Swanson

Authors
  1. Barb Thorne
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  2. Ryan Takeya
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  3. Francesca Vitelli
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  4. Xin Swanson
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Corresponding author

Correspondence to Xin Swanson .

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Editors and Affiliations

  1. Sutro Biopharma Inc., San Francisco, CA, USA

    Bob Kiss

  2. Lonza, Basel, Switzerland

    Uwe Gottschalk

  3. Solothurn Manufacturing Facility, Biogen International GmbH, Luterbach, Switzerland

    Michael Pohlscheidt

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Thorne, B., Takeya, R., Vitelli, F., Swanson, X. (2017). Gene Therapy. In: Kiss, B., Gottschalk, U., Pohlscheidt, M. (eds) New Bioprocessing Strategies: Development and Manufacturing of Recombinant Antibodies and Proteins. Advances in Biochemical Engineering/Biotechnology, vol 165. Springer, Cham. https://doi.org/10.1007/10_2016_53

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