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Gene and Cell Therapy: How to Build a BioDrug

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Book cover Pediatric Cancer Therapeutics Development

Part of the book series: Pediatric Oncology ((PEDIATRICO))

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Abstract

BioDrugs, or advanced therapeutic medicinal products (ATMP), are novel medicines involving genes, tissues, or cells for use in the treatment of a variety of diseases. The European Medicines Agency (EMA) classifies ATMP into three general categories: (1) gene therapies, (2) somatic-cell therapies, and (3) tissue-engineered medicines (https://www.ema.europa.eu/en/human-regulatory/overview/advanced-therapy-medicinal-products-overview), although there is overlap in the types of technologies used to create BioDrugs in these categories. For example, techniques used to edit or insert genes may be used to create a BioDrug used for gene therapy or for somatic-cell therapy. Development of a successful BioDrug requires in-depth knowledge of cellular biology and molecular genetics, complex manufacturing procedures, and completion of rigorous clinical trials in patients with serious medical illness. This chapter provides a resource for pediatric hematologist/oncologists to learn about the fundamental technologies involved in BioDrug development and will focus on the development of BioDrugs in the first two categories: (1) gene therapies, defined as a BioDrug that contain genes for insertion into the human genome or that contain gene-editing machinery for intracellular correction of genetic diseases, and (2) cell therapies, defined as cell products or tissues that have been manipulated to change their biologic characteristics with the aim to cure human disease.

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Authors and Affiliations

  1. Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA, USA

    Susanne Baumeister

  2. Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA

    Susanne Baumeister

  3. Harvard Medical School, Boston, MA, USA

    Susanne Baumeister

  4. Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA

    Ann Woolfrey

  5. Medpace, Inc., Cincinnati, OH, USA

    Ann Woolfrey

Authors
  1. Susanne Baumeister
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  2. Ann Woolfrey
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Correspondence to Ann Woolfrey .

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Editors and Affiliations

  1. Clinical Development, Kronos Bio, San Mateo, CA, USA

    Jorge DiMartino

  2. Department of Pediatrics, George Washington University, Washington, DC, USA

    Gregory H. Reaman

  3. Medpace, Cincinnati, OH, USA

    Franklin O. Smith

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Baumeister, S., Woolfrey, A. (2022). Gene and Cell Therapy: How to Build a BioDrug. In: DiMartino, J., Reaman, G.H., Smith, F.O. (eds) Pediatric Cancer Therapeutics Development. Pediatric Oncology. Springer, Cham. https://doi.org/10.1007/978-3-031-06357-2_6

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  • DOI: https://doi.org/10.1007/978-3-031-06357-2_6

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  • Online ISBN: 978-3-031-06357-2

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