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Current state of U.S. Food and Drug Administration regulation for cellular and gene therapy products: potential cures on the horizon

  • Michael Mendicino
    Correspondence
    Correspondence: Michael Mendicino, Ph.D. Chief Consultant & Advisor, Hybrid Concepts International (HCI), LLC; ARM Science & Technology Co-Chair; SCB Co-Founder and Member of Board of Directors. Mailing Address: 2135 Bedell Road, Suite 9b, Grand Island NY 14072.
    Affiliations
    Hybrid Concepts International (HCI), LLC

    Alliance for Regenerative Medicine (ARM) Science & Technology Committee

    Standards Coordinating Body for Gene, Cell, and Regenerative Medicines and Cell-based Drug Discovery (SCB)

    International Society for Cell & Gene Therapy North American Legal & Regulatory Affairs Committee
    Search for articles by this author
  • Yong Fan
    Affiliations
    A2Z Reg Solutions, LLC

    International Society for Cell & Gene Therapy North American Legal & Regulatory Affairs Committee
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  • Deborah Griffin
    Affiliations
    Chimera TQM LLC
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  • Kurt C. Gunter
    Affiliations
    Cell Medica, Inc.

    International Society for Cell & Gene Therapy North American Legal & Regulatory Affairs Committee
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  • Karen Nichols
    Affiliations
    Magenta Therapeutics, Inc.

    International Society for Cell & Gene Therapy North American Legal & Regulatory Affairs Committee
    Search for articles by this author

      Abstract

      Cellular & Gene Therapies (CGTs) are complex products, which have been key foci of the International Society for Cell & Gene Therapy (ISCT). For this ISCT North American Legal & Regulatory Affairs Committee review publication, CGTs include but are not limited to somatic cell-based therapies, pluripotent cell-derived cell-based therapies, gene- or non-gene-modified or gene edited versions of these cell-based therapies, in vivo gene therapies, organ/tissue engineered products, and relevant combination products. These products are regulated by the Food and Drug Administration (FDA) in the United States. This publication reviews selected laws, regulations, guidance, definitions, processes, types of meetings and submissions, and other key factors that the FDA follows and implements to regulate and support development of these types of products. These factors may be considered in order to help current and potential product developers/sponsors/applicants navigate through FDA regulatory pathways. We also review expedited programs including types of Designations available at the FDA, and their specific eligibility criteria. We include FDA and other stakeholder resources to consider regarding CGT regulation, to help prepare for CGT development and subsequent FDA approval.
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