Considerations for Cell and Gene Therapy Programs Entering the Clinical Space

Clin Pharmacol Ther. 2023 Sep;114(3):569-577. doi: 10.1002/cpt.2971. Epub 2023 Jun 25.

Abstract

Cell and gene therapy (CGT) describes a broad category of medicinal products with potential applications to prevent and treat human disease in multiple therapeutic areas. These therapies leverage the use of modified nucleic acids, altered cells or tissue, or both. The modality, mechanism, route of administration, and therapeutic indication for a CGT product will influence the challenges and opportunities for early clinical development, some of which may be highly specific to the product under consideration. Both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) encourage early interaction between sponsor and health authority to align on key elements of the CGT development program.

Publication types

  • Review

MeSH terms

  • Cell- and Tissue-Based Therapy*
  • Genetic Therapy*
  • Humans
  • United States
  • United States Food and Drug Administration