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Master Thesis

Enjeux réglementaires du développement des thérapies géniques dans l'Union européenne : l'exemple des cellules T à récepteur antigénique chimérique

Abstract : Over the past years, pharmaceutical research actors have put their efforts into the development of a new type of therapy using genes of interest to treat severe diseases. Gene therapies are a new class of medicines for which structural and functional features significantly differ from classic drugs. Their development involve genetic manipulation from which gene expression is directly responsible for the desired therapeutic effect. This is a major breakthrough compared to conventional medicines that pushed the European Medicine Agency to implement new regulatory tools: on the one hand to provide a framework for development and marketing of such therapies, and on the other hand to encourage and facilitate access to patients in need. This thesis is illustrated by the recent development and marketing authorisation in Europe of Kymriah® and Yescarta®, two chimeric antigen receptor T cells. These examples show the challenges raised by the emergence of these innovative therapies and depict the different regulatory strategies proposed by the European Medicine Agency to achieve their marketing authorisation.
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Master Thesis
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https://dumas.ccsd.cnrs.fr/dumas-03099938
Contributor : Ufr Santé Unicaen <>
Submitted on : Wednesday, January 6, 2021 - 1:39:02 PM
Last modification on : Friday, February 12, 2021 - 3:29:22 AM

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MASSON-Clementine.pdf
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  • HAL Id : dumas-03099938, version 1

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Clémentine Masson. Enjeux réglementaires du développement des thérapies géniques dans l'Union européenne : l'exemple des cellules T à récepteur antigénique chimérique. Sciences pharmaceutiques. 2020. ⟨dumas-03099938⟩

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