Comparative quality aspects of gene therapy medicinal products and medicinal products containing genetically modified cells

Aurélie Mahalatchimy (1) , Éloïse Gennet (1) , Véronique Andrieu (2) , Marion Feraud (3) , Victoire Lacaille (3) , Julie Veran (4) , Florence Sabatier (5) , Michael Morrison (6)

1 DICE / CERIC - Droits International, Comparé et Européen / Centre d'études et de recherches internationales et communautaires (CERIC)
2 MEPHI - Microbes évolution phylogénie et infections
3 AMU - Aix Marseille Université
4 APHM - Assistance Publique - Hôpitaux de Marseille
5 C2VN - Centre recherche en CardioVasculaire et Nutrition = Center for CardioVascular and Nutrition research
6 University of Oxford

The European Medicines Agency has recently revised its guidelines on quality, non-clinical and clinical aspects of Gene Therapy Medicinal Products (GTMPs text) and of medicinal products containing genetically modified cells (GMCMPs text), respectively in 2018 for the former and in 2020 for the latter. The latest revision has been particularly justified by the new developments in genome editing techniques, especially the wide use of the CRISPR-Cas technique, and with T-cells. Gene therapy medicinal product (GTMP) is a legal sub-category of medicines among Advanced Therapy Medicinal Products (ATMPs). However, medicinal product containing genetically modified cells (GMCMP) is not explicitly mentioned as a legal subcategory under EU regulation n°1394/2007 on ATMPs. Nevertheless, both GTMPs and GMCMPs should be considered as ATMPs under European Union Law. On the one hand, quality issues have been raised for all ATMPs regarding the reproducibility of the manufacturing process given the variable character of the starting materials. On the other hand, there is an overlap in the scope of the aforementioned guidelines when medicinal products containing genetically modified cells are to be considered gene therapy medicinal products. What are the similarities and differences between the quality of gene therapy medicinal products and the quality of medicinal products containing genetically modified cells while both are ATMPs? This poster is based on a comparative analysis of the quality sections of the two above-mentioned texts. In a first systematic approach, we identified the similarities and the specificities of texts outline (sub-section titles). Then, we analysed contents through the establishment of 3 types of comparative tables using color codes: common titles, different titles with similar contents, different titles with different contents in order to highlight similarities and specificities regarding the quality requirements between these two kinds of medicinal products when they are intended for use in humans and presented for marketing authorization applications.

Domaines: Droit

Aurélie Mahalatchimy, Éloïse Gennet, Véronique Andrieu, Marion Feraud, Victoire Lacaille, et al.. Comparative quality aspects of gene therapy medicinal products and medicinal products containing genetically modified cells. Annual Conference of the European Association of Health Law, Apr 2022, Ghent, Belgium. ⟨halshs-03779383⟩