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Advanced Therapies: Clinical, Non-clinical and Quality Considerations

  • Karin H. HoogendoornEmail author
Chapter

Abstract

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 of 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.

Keywords

Cell-based medicinal product Tissue engineered product Ex-vivo gene therapy Clinical considerations Non-clinical considerations Production and testing considerations Regulatory considerations 

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Suggested Reading

  1. BSI PAS 83:2012 (2012) Developing human cells for clinical applications in the Euopean Union and the United States of America – guide. Publicly Available Specification PAS83:2012, The British Standards Institution, ISBN 978 0 580 71052 0Google Scholar
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Copyright information

© Springer Nature Switzerland AG 2019

Authors and Affiliations

  1. 1.Leiden University Medical Center, Hospital Pharmacy, Interdivisional GMP FacilityLeidenThe Netherlands

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