Regulatory Progress in the Clinical Translation of Gene Therapy in China: Evolution, Challenges, and Future Perspectives

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’s 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 “818 Regulation”). 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.