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Advanced Therapy Medicinal Products: Clinical, Non-clinical, and Quality Considerations

  • Enrico Mastrobattista,
  • Erik Doevendans,
  • Niek P. van Til,
  • Vera Kemp,
  • Jeroen de Vrij,
  • Karin Hoogendoorn

摘要

Cell therapy, tissue engineering, and gene therapy products, together called “advanced therapy medicinal products” (ATMPs), represent a heterogeneous group of innovative biopharmaceuticals. ATMPs are based on viable cells, tissue, or genetic material. In this chapter, after a brief introduction, first different classification systems for these products are discussed, illustrated with representative examples of products in clinical development or commercially available. Next, the challenges associated with successful pharmaceutical development, manufacturing, and testing of these products are covered. Finally, regulatory aspects are dealt with.