If the United States and Germany remain leaders in preclinical research on CAR-T therapies, China is now leading in their clinical development, according to a recent analysis. This progress is primarily attributed to accelerated and more flexible procedures, as well as significant government support in terms of funding and regulation. CAR-T cell therapy is an immunotherapy that involves modifying the patient's immune cells to direct them specifically toward their tumor or defective cells. It comes in two forms: in vivo CAR-T and ex vivo CAR-T. The first involves encouraging the patient's body to modify its immune cells from within, while the second involves extracting these cells, modifying them in the laboratory, and then reintroducing them into the patient's body. In vivo CAR-T could potentially reduce costs and simplify the manufacturing process compared to ex vivo CAR-T, and both are already being explored for various conditions such as blood cancer, liver disease, and lupus.
In an analysis published last month in Nature Reviews Drug Discovery, researchers from the Boston Consulting Group (Massachusetts) revealed that most of the fundamental preclinical work in the field of CAR-T therapies has been conducted in the United States and Germany. However, China has, according to them, rapidly advanced toward clinical trials. Of the 140 in vivo CAR-T therapy programs at the preclinical or clinical stage worldwide, 82 percent are being developed by Chinese teams, according to the analysis. These results show that China holds an important position in the clinical development of in vivo CAR-T therapies. "Successful clinical development in China could also accelerate innovation, reduce treatment costs, and generate valuable clinical data that would benefit patients worldwide," explains Xiaoyuan Chen, a chemist at the hospital and institute of cancer in Shandong, Jinan, China, who is developing an in vivo CAR-T therapy for cancer and fibrosis.
The report indicates that the first results of clinical trials conducted in China are encouraging. The results of the first human trial of an in vivo CAR-T therapy for lupus were published in September 2025. Five patients with a resistant form of the disease showed improvement in their symptoms only three months after treatment, without serious side effects. In September 2026, Chinese researchers also reported clinical improvement in 16 people with refractory neurological autoimmune diseases after an in vivo CAR-T cell therapy. Other results are expected in the coming years for autoimmune diseases and cancer. According to John Wu from the Boston Consulting Group, a co-author of the analysis, these advances are attributed to the substantial institutional funding that CAR-T therapy research in China benefits from. The work also benefits from close collaboration between universities, hospitals, and pharmaceutical laboratories and biotechnology companies.
The country also has an investigator-initiated trial (IIT) system, which allows for rapid testing of certain experimental therapies on humans and the production of preliminary clinical data without direct supervision from the national regulatory authority. Combined with the size of China's population and the number of patients with autoimmune diseases who have few therapeutic alternatives, the IIT makes clinical trials much less expensive. According to John Wu, a comparable trial could cost about half as much in China as in the United States. The regulatory facilitations seem to affect the clinical trials of in vivo CAR-T, the researchers specified. According to them, at least six in vivo CAR-T trials have been suspended or withdrawn since May in order to comply with new regulatory requirements. On the other hand, the long-term benefits and safety of this therapy remain to be demonstrated before considering its large-scale use. Nevertheless, this work could also have repercussions beyond China. This month, the United States Food and Drug Administration (FDA) approved the initiation of a clinical trial for the first Chinese-origin in vivo CAR-T therapy involved in this procedure, intended for the treatment of blood cancers. Other international groups also show interest in collaborating with China for the development of other CAR-T therapies.
China Leads in Clinical Development of CAR-T Therapies, Analysis Shows
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