Sanchez-Guijo, Fermin and Vives, Joaquim and Ruggeri, Annalisa and Chabannon, Christian and Corbacioglu, Selim and Dolstra, Harry and Farge, Dominique and Gagelmann, Nico and Horgan, Claire and Kuball, Jurgen and Neven, Benedicte and Rintala, Tuula and Rocha, Vanderson and Sanchez-Ortega, Isabel and Snowden, John A. and Zwaginga, Jaap Jan and Gnecchi, Massimiliano and Sureda, Anna (2024) Current challenges in cell and gene therapy: a joint view from the European Committee of the International Society for Cell & Gene Therapy (ISCT) and the European Society for Blood and Marrow Transplantation (EBMT). CYTOTHERAPY, 26 (7). pp. 681-685. ISSN 1465-3249, 1477-2566
Full text not available from this repository. (Request a copy)Abstract
Cell and gene therapy poses evolving challenges. The current article summarizes the discussions held by European Regional Committee of the International Society for Cell & Gene Therapy and the European Society for Blood and Marrow Transplantation (EBMT) on the current challenges in this field, focusing on the European setting. This article emphasizes the imperative assessment of real -world cell and gene therapy activity, advocating for expanded registries beyond hematopoietic transplantation and chimeric antigen receptor -Tcell therapy. Accreditation 's role in ensuring standardized procedures, as exemplified by JACIE (The Joint Accreditation Committee of ISCT-Europe and EBMT), is crucial for safety. Access to commercial products and reimbursement variations among countries underscore the need for uniform access to advanced therapy medical products (ATMPs). Academic product development and point -of -care manufacturing face barriers to patient access. Hospital Exemption 's potential, demonstrated by some initial experiences, may increase patient accessibility in individual situations. Regulatory challenges, including the ongoing European ATMPs legislation review, necessitate standardized criteria for Hospital Exemption and mandatory reporting within registries. Efforts to combat unproven therapies and fraud involve collaboration between scientific societies, regulatory bodies and patient groups. Finally, is important to highlight the vital role of education and work- force development in meeting the escalating demand for specialized professionals in the ATMP field. Collabo- ration among scientific societies, academic institutions, industry, regulatory bodies and patient groups is crucial for overcoming all these challenges to increase gene and cell therapy activity in Europe. (c) 2024 International Society for Cell & Gene Therapy. Published by Elsevier Inc. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/)
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | ENGINEERED TISSUE THERAPIES; ATMPs; benchmarking; CAR-T; cell and gene therapy; EBMT; education; EMA; Europe; ISCT; JACIE; MSC; registry; reimbursement |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 18 Aug 2025 08:20 |
| Last Modified: | 18 Aug 2025 08:20 |
| URI: | https://pred.uni-regensburg.de/id/eprint/65640 |
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