Yang Yifan, Bian Lianlian, Cheng Yuan, Xu Yan, Shao Hui, Rao Jian, Ge Sixiang, Gong Jifang, Jiang Min, Zheng Xiaoyu, Liu Lijun, Ma Shihui, Liu Xuan, Cheng Tao, Gao Chenyan
Changping Laboratory, Beijing, China.
China Pharmaceutical University, Nanjing, China.
Clin Transl Sci. 2025 Feb;18(2):e70148. doi: 10.1111/cts.70148.
As cutting-edge technologies in biomedicine, cell and gene therapy (CGT) products demonstrate immense potential in treating cancer, rare diseases, and genetic disorders, thereby driving the importance of clinical research in this area. This study analyzes the growth trends and key characteristics of 1033 Investigator-Initiated Trials (IITs) conducted by mainland Chinese institutions in the CGT field. The results show that IITs have played a positive role in the early proof-of-concept of CGT products, helping to obtain preliminary safety and efficacy data, and exploring the combination of CGT products with other therapies. Additionally, this study discusses the regional distribution, therapeutic areas, and challenges faced by IITs in the development of CGT products in China. Based on these findings, policy suggestions are proposed to optimize the regulation of IITs in mainland China, such as improving regulatory frameworks and enhancing technical guidance. It is hoped that these measures will further improve the efficiency and quality of IITs, fully utilize the large patient base and abundant clinical resources, and support the development of high-quality CGT products in mainland China.
作为生物医学领域的前沿技术，细胞和基因疗法（CGT）产品在治疗癌症、罕见病和遗传性疾病方面展现出巨大潜力，从而凸显了该领域临床研究的重要性。本研究分析了中国大陆机构在CGT领域开展的1033项研究者发起的试验（IIT）的增长趋势和关键特征。结果表明，IIT在CGT产品的早期概念验证中发挥了积极作用，有助于获取初步的安全性和有效性数据，并探索CGT产品与其他疗法的联合应用。此外，本研究还讨论了中国IIT在CGT产品研发中的区域分布、治疗领域以及面临的挑战。基于这些发现，提出了优化中国大陆IIT监管的政策建议，如完善监管框架和加强技术指导。希望这些措施将进一步提高IIT的效率和质量，充分利用庞大的患者群体和丰富的临床资源，支持中国大陆高质量CGT产品的研发。
