Center for Drug Evaluation (CDE), National Medical Products Administration, Beijing, 100022, China.
Center for Drug Evaluation (CDE), National Medical Products Administration, Beijing, 100022, China.
Biologicals. 2020 Nov;68:3-8. doi: 10.1016/j.biologicals.2020.10.001. Epub 2020 Oct 20.
Tremendous progress has been made in recent years to produce functional cells for cell therapy products. Hundreds of clinical trials of stem cell products (SCPs) have shown promising therapeutic potential worldwide, including the products derived from human pluripotent stem cells (hPSCs), adult stem cells and mesenchymal stem cells (MSC). Before starting a clinical trial, comprehensive chemistry, manufacturing and control (CMC) study is required to assure the safety and quality consistency of SCPs. The heterogeneity of stem cell products arises from the variability in the donor tissues, isolation of cells and differentiation processes, and appropriate testing approaches are needed to characterize and release SCPs. Here we summarize the regulatory considerations of CMC study in Investigational New Drug (IND) application of SCPs in China based on the current knowledge, and they will be updated in the future with the advance of stem cell biology and regulatory science.
近年来，在生产用于细胞治疗产品的功能性细胞方面取得了巨大进展。全球数百项干细胞产品（SCP）的临床试验显示出了有前景的治疗潜力，其中包括源自人类多能干细胞（hPSC）、成体干细胞和间充质干细胞（MSC）的产品。在开始临床试验之前，需要进行全面的化学、制造和控制（CMC）研究，以确保 SCP 的安全性和质量一致性。干细胞产品的异质性源于供体组织、细胞分离和分化过程的可变性，需要适当的测试方法来对 SCP 进行表征和放行。在这里，我们根据目前的知识总结了中国 SCP 的新药临床试验（IND）申请中 CMC 研究的监管考虑因素，随着干细胞生物学和监管科学的进步，这些考虑因素将在未来得到更新。
