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Review
. 2024 Jul;26(7):681-685.
doi: 10.1016/j.jcyt.2024.02.007. Epub 2024 Feb 17.

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)

Fermin Sanchez-Guijo  1 , Joaquim Vives  2 , Annalisa Ruggeri  3 , Christian Chabannon  4 , Selim Corbacioglu  5 , Harry Dolstra  6 , Dominique Farge  7 , Nico Gagelmann  8 , Claire Horgan  9 , Jurgen Kuball  10 , Benedicte Neven  11 , Tuula Rintala  12 , Vanderson Rocha  13 , Isabel Sanchez-Ortega  12 , John A Snowden  14 , Jaap Jan Zwaginga  15 , Massimiliano Gnecchi  16 , Anna Sureda  17
Affiliations
Free article
Review

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)

Fermin Sanchez-Guijo et al. Cytotherapy. 2024 Jul.
Free article

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-T-cell 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 workforce development in meeting the escalating demand for specialized professionals in the ATMP field. Collaboration 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.

Keywords: ATMPs; CAR-T; EBMT; EMA; Europe; ISCT; JACIE; MSC; benchmarking; cell and gene therapy; education; registry; reimbursement.

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Conflict of interest statement

Declaration of Competing Interest FSG, JV, DF, JJZ and MG are members of the International Society for Cellular Therapy (ISCT-EU) Executive Committee. AR, CC, SC, HD, NG, CH, JHEK, BN, TR, ISG, JAS and AS are members of European Society for Blood and Marrow Transplantation (EBMT) executive committee or working groups representatives. FSG has received research support from Novartis, Gilead. Honoraria from Novartis, Gilead, Pfizer, BMS-Celgene and Pierre-Fabré. CC has received honoraria (personal and institutional) and travel support from Bellicum Pharmaceuticals, BMS, Jazz Pharmaceuticals, Kite / Gilead, Novartis and Sanofi SA as a compensation for speaker's bureau and advisory boards. JK was shareholder of Gadeta and is inventor on multiple patents dealing with engineered immune cells, and has received research support from Novartis, Milteny Biotech and Gadeta. JAS has received consultancy honoraria from Kiadis, Medac, Vertex and Jazz. AS has received research support from Takeda and honoraria from Takeda, BMS/Celgene, MSD, Novartis, Gilead Kite, Sanofi, Pierre Fabre, Janssen and Jazz Pharmaceuticals. All other authors have no commercial, proprietary or financial interest in the products or companies described in this article.