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Overview of Chemistry, Manufacturing, and Controls (CMC) for Pluripotent Stem Cell-Based Therapies

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Regenerative Medicine for Degenerative Muscle Diseases

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

Abstract

The US Food and Drug Administration (FDA) regulates pluripotent stem cell (PSC)-based therapies under guidelines that apply to Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps). In this chapter, we will examine the various components necessary to generate a rigorous Chemistry, Manufacturing, and Controls (CMC) section for an FDA Investigational New Drug (IND) application for these products. Our discussion begins with regulations that apply to product components and materials used during the manufacturing process and then proceeds to facilities, procedures, personnel, and process controls required for implementing Good Tissue Practice (GTP) into current Good Manufacturing Procedures (cGMP). Product testing, methods to establish product stability, and logistical details including labeling, packaging, and postproduction requirements will also be described. Finally, quality systems for manufacture and distribution of investigational products will be presented.

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Authors and Affiliations

  1. Regenerative Medicine Strategy Group, LLC, 10401 Venice Boulevard, Suite 354, Los Angeles, CA, 90034, USA

    Amy Lynnette Van Deusen & Michael Earl McGary

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  1. Amy Lynnette Van Deusen
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  2. Michael Earl McGary
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Correspondence to Amy Lynnette Van Deusen .

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

    Martin K. Childers

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Van Deusen, A.L., McGary, M.E. (2016). Overview of Chemistry, Manufacturing, and Controls (CMC) for Pluripotent Stem Cell-Based Therapies. 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_7

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