{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T04:50:22Z","timestamp":1791435022678,"version":"4.3.3"},"reference-count":12,"publisher":"SAGE Publications","issue":"15-16","license":[{"start":{"date-parts":[[2026,7,6]],"date-time":"2026-07-06T00:00:00Z","timestamp":1783296000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,7,6]],"date-time":"2026-07-06T00:00:00Z","timestamp":1783296000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2026,8,1]]},"abstract":"<jats:p>Gene- and cell-based therapies (GCTs) represent a disruptive and transformative class of biomedical innovations. They address diseases by adding, removing, repairing, or replacing genes and\/or by endowing distinct living cells with additional biological functions. Through this plethora of options, numerous conditions\u2014including genetic disorders, cancers, and degenerative diseases\u2014have become potential targets for a curative therapy. Thus, GCTs are considered the \u201cFuture of Medicine\u201d as they (i) offer a potential cure, particularly for rare and severe disorders previously considered untreatable, (ii) expand the treatment options for common diseases, and (iii) possess the possibility to complement currently applied conventional treatment options. Recognizing both the scientific promise and translational challenges of GCTs, Germany has launched a coordinated national initiative\u2014the National Strategy for Gene- and Cell-Based Therapies. The Strategy was commissioned by the German Federal Ministry of Research, Technology and Space (BMFTR, formerly the German Federal Ministry of Education and Research [BMBF]) and developed through a multi-stakeholder process. The latter involved more than 150 experts from academia, industry, health care sector, professional associations, and patient organizations, who were nominated by the community and assembled into eight working groups to identify current roadblocks and propose possible solutions. Summarized in the Strategy Paper, which was submitted to the BMFTR and published on June 12, 2024, a comprehensive roadmap was developed in this bottom-up process to accelerate the development and clinical implementation of GCTs in Germany. Although it initially had a national focus, the resulting framework is increasingly contributing to the international GCT landscape through growing exchange with GCT initiatives launched in other European member states and with the European Society of Gene and Cell Therapy (ESGCT).<\/jats:p>\n                  <jats:p>In brief, the initiative is focusing on translation starting from research through all steps to clinical application and beyond. This includes workforce development, regulatory frameworks, manufacturing capacity, patient access, and communication with the general public. Numerous targeted measures have been developed by the participating experts in the working groups and are currently being implemented in this broad, collaborative, and bottom-up multi-stakeholder approach. They encompass, for example, the establishment of a website as central information platform, including the GCT-Atlas, a web-based networking and information tool for stakeholders and actors in the GCT field, tailored communication and outreach formats, a Regulatory Support Unit providing independent regulatory guidance for publicly funded early-stage, nonclinical product development, different funding and entrepreneurship programs offering researchers and clinicians financial, educational, and mentoring support, as well as the establishment of translational infrastructure and exchange formats with investors to specifically foster the necessary scale-up and commercialization.<\/jats:p>\n                  <jats:p>Overall, the main goal of the German National Strategy for GCT is to ensure patient access to advanced therapies while strengthening Germany\u2019s position as an international hub for biomedical innovation. To accomplish this, existing resources need to be coordinated, streamlined, and prioritized to increase efficiency and support the long-term sustainability of the system. These objectives are closely aligned with current emerging European initiatives, including the EU Biotech Act and the Horizon Europe work program 2026, which aim to further optimize the framework conditions for this strategically important field and enhance future European competitiveness.<\/jats:p>","DOI":"10.1177\/10430342261464845","type":"journal-article","created":{"date-parts":[[2026,7,7]],"date-time":"2026-07-07T06:48:11Z","timestamp":1783406891000},"page":"623-629","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["The German National Strategy for Gene- and Cell-Based Therapies: Generating Impact by Employing a Novel Multi-Stakeholder Approach"],"prefix":"10.1177","volume":"37","author":[{"ORCID":"https:\/\/orcid.org\/0009-0008-6381-7639","authenticated-orcid":false,"given":"Christian","family":"Gallus","sequence":"first","affiliation":[{"name":"Strategic Science Management, Berlin Institute of Health at Charit\u00e9, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francis","family":"Ayuk","sequence":"additional","affiliation":[{"name":"Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philipp","family":"Beckhove","sequence":"additional","affiliation":[{"name":"Leibniz Institute for Immunotherapy, Regensburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Besser","sequence":"additional","affiliation":[{"name":"Dialog Platform Stem Cell Research, Berlin Institute of Health at Charit\u00e9, Berlin, Germany"},{"name":"German Stem Cell Network eV, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Bornh\u00e4user","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Dresden University Hospital, Dresden, Germany"},{"name":"National Center for Tumor Diseases Dresden, Dresden, Germany"},{"name":"Cluster4Future SaxoCell, Dresden, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3365-6933","authenticated-orcid":false,"given":"Hildegard","family":"B\u00fcning","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany"},{"name":"REBIRTH Research Center for Translational Regenerative Medicine, Hannover Medical School, Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tobias","family":"Cantz","sequence":"additional","affiliation":[{"name":"REBIRTH Research Center for Translational Regenerative Medicine, Hannover Medical School, Hannover, Germany"},{"name":"Department of Gastroenterology, Hepatology, Infectious Diseases and Endocrinology, Hannover Medical School, Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christoph","family":"Conrad","sequence":"additional","affiliation":[{"name":"Regulatory Support Unit, Berlin Institute of Health at Charit\u00e9, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefan","family":"Eichm\u00fcller","sequence":"additional","affiliation":[{"name":"Research Group GMP &amp; T Cell Therapy, German Cancer Research Center, Heidelberg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Gazlig","sequence":"additional","affiliation":[{"name":"Department of Charit\u00e9 BIH Innovation, Charit\u00e9 \u2013 Universit\u00e4tsmedizin Berlin and Berlin Institute of Health at Charit\u00e9, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Holm","family":"Graessner","sequence":"additional","affiliation":[{"name":"Institute of Medical Genetics and Applied Genomics, Universit\u00e4tsklinikum T\u00fcbingen, T\u00fcbingen, Germany"},{"name":"Centre for Rare Diseases, Universit\u00e4tsklinikum T\u00fcbingen, T\u00fcbingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annette","family":"Gr\u00fcters-Kieslich","sequence":"additional","affiliation":[{"name":"Eva Luise and Horst K\u00f6hler Foundation for Rare Diseases, Bonn, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathalie","family":"Huber","sequence":"additional","affiliation":[{"name":"Biomedical Innovation Academy, Berlin Institute of Health at Charit\u00e9, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Hudecek","sequence":"additional","affiliation":[{"name":"Department of Medicine II, Universit\u00e4tsklinikum W\u00fcrzburg, W\u00fcrzburg, Germany"},{"name":"Fraunhofer Institute for Cell Therapy and Immunology IZI, Leipzig, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thekla","family":"Kurz","sequence":"additional","affiliation":[{"name":"Bioindustry Division, Miltenyi Biotec BV &amp; Co KG, Bergisch Gladbach, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bj\u00f6rn","family":"Lamprecht","sequence":"additional","affiliation":[{"name":"Commercial Excellence, Roche Pharma AG, Grenzach-Wyhlen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elke","family":"Luger","sequence":"additional","affiliation":[{"name":"National Network Office GCT, Berlin Institute of Health at Charit\u00e9, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ulrike","family":"Protzer","sequence":"additional","affiliation":[{"name":"Institute of Virology, Technical University of Munich School of Medicine and Helmholtz Center Munich German Research Center for Environmental Health, Neuherberg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Axel","family":"Schambach","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany"},{"name":"REBIRTH Research Center for Translational Regenerative Medicine, Hannover Medical School, Hannover, Germany"},{"name":"Division of Hematology\/Oncology, Boston Children\u2019s Hospital, Boston, Massachusetts, USA"},{"name":"Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Sch\u00e4fer","sequence":"additional","affiliation":[{"name":"Institute for Transfusion Medicine and Gene Therapy, Medical Center \u2013 University of Freiburg, Freiburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pablo","family":"Serrano","sequence":"additional","affiliation":[{"name":"Innovation and Research\/Biotechnology, German Pharmaceutical Industry Association, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sven","family":"Stegemann","sequence":"additional","affiliation":[{"name":"Leibniz JointLab First in Translation, Leibniz Institute for Interactive Materials, Aachen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabelle","family":"St\u00f6ckert","sequence":"additional","affiliation":[{"name":"Independent Regulatory Expert, Bonn, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christof","family":"von Kalle","sequence":"additional","affiliation":[{"name":"Clinical Study Center, Berlin Institute of Health at Charit\u00e9, Berlin, Germany"},{"name":"Luxembourg Research Clinic (LRC)\u2014Fuerschungsklinik L\u00ebtzebuerg, Luxemburg Institute of Health, Luxembourg, Luxemburg"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joachim","family":"Weber","sequence":"additional","affiliation":[{"name":"Strategic Science Management, Berlin Institute of Health at Charit\u00e9, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Baum","sequence":"additional","affiliation":[{"name":"Berlin Institute of Health at Charit\u00e9, Berlin, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,7,6]]},"reference":[{"key":"e_1_3_2_2_1","unstructured":"1.Berlin Institute of Health at Charit\u00e9 \u2013 Universit\u00e4tsmedizin Berlin (BIH). National Strategy for Gene- and Cell-based Therapies. 2024. Available from: https:\/\/www.nationale-strategie-gct.de\/fileadmin\/gct\/01_Ueber\/NEU_250619_Strategiepapier_EN.pdf"},{"key":"e_1_3_2_3_1","unstructured":"2.Berlin Institute of Health at Charit\u00e9 \u2013 Universit\u00e4tsmedizin Berlin (BIH). National Strategy for Gene- and Cell-based Therapies. 2025. Available from: https:\/\/www.nationale-strategie-gct.de\/en\/"},{"key":"e_1_3_2_4_1","unstructured":"3.Berlin Institute of Health at Charit\u00e9 \u2013 Universit\u00e4tsmedizin Berlin (BIH). The GCT Atlas. 2025. Available from: https:\/\/www.nationale-strategie-gct.de\/en\/national-network-office\/overview\/"},{"key":"e_1_3_2_5_1","unstructured":"4.Berlin Institute of Health at Charit\u00e9 \u2013 Universit\u00e4tsmedizin Berlin (BIH). Working Goups of the National Strategy for Gene- and Cell-Based Therapies. 2024. 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Available from: https:\/\/www.nationale-strategie-gct.de\/en\/funding\/personal-funding\/"},{"key":"e_1_3_2_9_1","unstructured":"8.Berlin Institute of Health at Charit\u00e9 \u2013 Universit\u00e4tsmedizin Berlin (BIH). Regulatory Support Unit (RSU). 2025. Available from: https:\/\/www.nationale-strategie-gct.de\/en\/regulatory-support-unit\/"},{"key":"e_1_3_2_10_1","unstructured":"9.Berlin Institute of Health at Charit\u00e9 \u2013 Universit\u00e4tsmedizin Berlin (BIH). GeneNovate\u00ae Investors\u2019 Day. 2025. Available from: https:\/\/www.nationale-strategie-gct.de\/en\/genenovate\/genenovate-investors-day\/"},{"key":"e_1_3_2_11_1","unstructured":"10.Gene and Cell Therapies Incubator Berlin GmbH. The Berlin Center for Gene and Cell Therapies. 2025. Available from: https:\/\/www.bc-gct.com\/"},{"key":"e_1_3_2_12_1","unstructured":"11.National Registry for Gene and Cell Therapies German. National Registry for Gene and Cell Therapies Germany. 2026. 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