{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T03:00:04Z","timestamp":1790391604845,"version":"4.1.0"},"reference-count":25,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2026,9,21]],"date-time":"2026-09-21T00:00:00Z","timestamp":1789948800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"name":"Jiangxi Provincial Management Science Program"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"abstract":"<jats:title\/>\n                  <jats:p>In recent years, gene therapy has emerged as a major frontier in biomedicine, with translation from basic research to clinical application accelerating worldwide. Clinical research in this field has also expanded substantially in China. Prior to the implementation of the new regulatory framework in 2026, investigator-initiated clinical research played an important role in early clinical exploration and technology validation, alongside product-oriented clinical trials conducted under the pharmaceutical regulatory pathway. However, under the previous framework, the pathways for translating research outcomes into clinical use and their interface with product development remained insufficiently defined. Against this background, this review systematically examines the evolution of China\u2019s regulatory framework for gene therapy clinical research and translation, with particular attention to the institutional changes introduced by the Regulation on the Administration of Clinical Research and Translational Application of New Biomedical Technology (the \u201c818 Regulation\u201d). Relevant regulatory approaches in the United States, the European Union, Japan, and South Korea are also examined for comparative context. The 818 Regulation provides a more clearly defined institutional framework for gene therapy clinical research and translation, while also introducing new practical requirements for research implementation. In particular, initiating institutions and clinical research institutions need to strengthen their professional implementation capacity and cross-departmental coordination. For innovative technologies at an early stage of development, where the ultimate translational direction may not yet be fully determined, greater attention should be given to the assessment of technological characteristics and prospective planning of translational pathways. Differences in data standards, quality-control requirements, and evidentiary expectations across translational pathways also place greater demands on evidence continuity and the future usability of research data. Accordingly, under the new regulatory environment, initiating institutions and clinical research institutions should strengthen internal governance and professional management, prospectively assess technological characteristics and intended translational objectives, appropriately plan research and translational pathways, and adopt high-standard data governance to improve the completeness, reliability, and traceability of research evidence. Such efforts would preserve opportunities for future data linkage and reuse, as well as the potential acceptability of data across different translational pathways. Effective institutional adaptation to the new framework will be critical to realizing the potential benefits of the 818 Regulation and improving the quality and efficiency of gene therapy clinical translation in China.<\/jats:p>","DOI":"10.1177\/10430342261490050","type":"journal-article","created":{"date-parts":[[2026,9,22]],"date-time":"2026-09-22T05:11:16Z","timestamp":1790053876000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Regulatory Progress in the Clinical Translation of Gene Therapy in China: Evolution, Challenges, and Future Perspectives"],"prefix":"10.1177","author":[{"given":"Kaizhen","family":"Wang","sequence":"first","affiliation":[{"name":"China National Center for Biotechnology Development, Beijing, China"},{"name":"School of Biomedical Engineering, Tsinghua University, Beijing, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nvjin","family":"Zeng","sequence":"additional","affiliation":[{"name":"Department of Science and Technology, The First Affiliated Hospital, Jiangxi Medical College, Nanchang University, Nanchang, Jiangxi, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hualan","family":"Yang","sequence":"additional","affiliation":[{"name":"Department of Neurology, Shanghai Pudong Hospital, Fudan University Pudong Medical Center, Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rui","family":"He","sequence":"additional","affiliation":[{"name":"China National Center for Biotechnology Development, Beijing, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qi","family":"Chen","sequence":"additional","affiliation":[{"name":"China National Center for Biotechnology Development, Beijing, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mingjin","family":"Jiang","sequence":"additional","affiliation":[{"name":"Department of Science and Technology, The First Affiliated Hospital, Jiangxi Medical College, Nanchang University, Nanchang, Jiangxi, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ping","family":"Liu","sequence":"additional","affiliation":[{"name":"China National Center for Biotechnology Development, Beijing, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yue","family":"Qiu","sequence":"additional","affiliation":[{"name":"School of Healthcare Management, Tsinghua Medicine, Tsinghua University, Beijing, China."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-0764-1812","authenticated-orcid":false,"given":"Zhenhang","family":"Yu","sequence":"additional","affiliation":[{"name":"China National Center for Biotechnology Development, Beijing, China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,9,21]]},"reference":[{"key":"e_1_3_4_2_1","volume-title":"Human Gene Therapy for Rare Diseases: Guidance for Industry","author":"U.S. Food and Drug Administration","year":"2020","unstructured":"1.U.S. Food and Drug Administration. 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Regulation on the Administration of Clinical Research and Translational Application of new biomedical technology. State Council Decree No. 818. 2025. Available from: https:\/\/www.gov.cn\/zhengce\/content\/202510\/content_7043790.htm [Last accessed: August12 2026]."},{"issue":"7","key":"e_1_3_4_11_1","first-page":"511","article-title":"Challenges of investigator-initiated clinical trials to support the new drug development","volume":"25","author":"Hua B","year":"2022","unstructured":"10.Hua B, , Zhang S. Challenges of investigator-initiated clinical trials to support the new drug development. Chin J Lung Cancer 2022;25(7):511\u2013516.","journal-title":"Chin J Lung Cancer"},{"key":"e_1_3_4_12_1","unstructured":"11.National Health Commission of the People\u2019s Republic of China National Administration of Traditional Chinese Medicine and National Disease Control and Prevention Administration. Administrative Measures for Investigator-Initiated Clinical Research Conducted by Medical and Health Institutions. Guo Wei Ke Jiao Fa [2024] No. 32. September 18 2024. Available from: https:\/\/www.nhc.gov.cn\/qjjys\/c100016\/202409\/3a3ad0a7b656420d9580b65f2321a623.shtml [Last accessed: August12 2026]."},{"key":"e_1_3_4_13_1","unstructured":"12.Grand View Research. China cell and gene therapy manufacturing market size and outlook. 2023. Available from: https:\/\/www.grandviewresearch.com\/horizon\/outlook\/cell-and-gene-therapy-manufacturing-market\/china"},{"key":"e_1_3_4_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/cts.70148"},{"issue":"11","key":"e_1_3_4_15_1","first-page":"746","article-title":"Analysis of common quality problems and countermeasures of clinical trials initiated by investigator","volume":"40","author":"Hu T","year":"2021","unstructured":"14.Hu T, , Lu W, , Shen E, et al. Analysis of common quality problems and countermeasures of clinical trials initiated by investigator. Chin J New Drugs Clin Remedies 2021;40(11):746\u2013749.","journal-title":"Chin J New Drugs Clin Remedies"},{"key":"e_1_3_4_16_1","first-page":"235","article-title":"The international experiences of investigator-initiated clinical trials and implications","author":"Zhang H","year":"2021","unstructured":"15.Zhang H, , Zhang B, , Yu L, et al. The international experiences of investigator-initiated clinical trials and implications. Chin J Med Sci Res Manag 2021:235\u2013240.","journal-title":"Chin J Med Sci Res Manag"},{"key":"e_1_3_4_17_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1706910"},{"key":"e_1_3_4_18_1","unstructured":"17.National Health Commission of the People\u2019s Republic of China. Interim Guiding Principles for the Classification of new biomedical technology Pharmaceuticals and Medical Devices. April 28 2026. Available from: https:\/\/www.nhc.gov.cn"},{"key":"e_1_3_4_19_1","volume-title":"Instructions for Filling Out Form FDA 1571: Investigational New Drug Application (IND)","author":"U.S. Food and Drug Administration","unstructured":"18.U.S. Food and Drug Administration. Instructions for Filling Out Form FDA 1571: Investigational New Drug Application (IND). U.S. Food and Drug Administration: Silver Spring, MD."},{"key":"e_1_3_4_20_1","volume-title":"Human Gene Therapy Products Incorporating Human Genome Editing: Guidance for Industry","author":"U.S. Food and Drug Administration","year":"2024","unstructured":"19.U.S. Food and Drug Administration. Human Gene Therapy Products Incorporating Human Genome Editing: Guidance for Industry. U.S. Food and Drug Administration: Silver Spring, MD; 2024."},{"key":"e_1_3_4_21_1","volume-title":"Guideline on the Quality, Non-clinical and Clinical Requirements for Investigational Advanced Therapy Medicinal Products in Clinical Trials","author":"European Medicines Agency","year":"2025","unstructured":"20.European Medicines Agency. Guideline on the Quality, Non-clinical and Clinical Requirements for Investigational Advanced Therapy Medicinal Products in Clinical Trials. EMA\/CAT\/22473\/2025. European Medicines Agency: Amsterdam; 2025."},{"key":"e_1_3_4_22_1","volume-title":"Guideline on the Quality, Non-clinical and Clinical Aspects of Gene Therapy Medicinal Products","author":"European Medicines Agency","year":"2018","unstructured":"21.European Medicines Agency. Guideline on the Quality, Non-clinical and Clinical Aspects of Gene Therapy Medicinal Products. EMA\/CAT\/80183\/2014. 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Ministry of Government Legislation: Seoul."},{"key":"e_1_3_4_26_1","volume-title":"Interim Administrative Measures for Clinical Research Projects in Medical and Health Institutions","author":"National Health and Family Planning Commission of the People\u2019s Republic of China","year":"2014","unstructured":"25.National Health and Family Planning Commission of the People\u2019s Republic of China. Interim Administrative Measures for Clinical Research Projects in Medical and Health Institutions. 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