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

  • Amy Lynnette Van Deusen
  • Michael Earl McGary
Chapter
Part of the Stem Cell Biology and Regenerative Medicine book series (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.

Keywords

CFR part 1271 Chemistry, manufacturing, and controls CMC Code of federal regulations Current good manufacturing practice cGMP FDA guidance for industry Good laboratory practice GLP Good tissue practice GTP Human cells, tissues and cellular, and tissue-based product HCT/P Induced pluripotent stem cells iPSC Investigational new drug application IND Pluripotent stem cells PSC 

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Copyright information

© Springer Science+Business Media New York 2016

Authors and Affiliations

  • Amy Lynnette Van Deusen
    • 1
  • Michael Earl McGary
    • 1
  1. 1.Regenerative Medicine Strategy Group, LLCLos AngelesUSA

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