China has emerged as the leading centre for clinical development and testing of a new generation of CAR-T-cell therapies that could eventually be used against cancer and autoimmune diseases, according to an analysis reported by Nature.
The therapies, known as in vivo CAR-T, are designed to modify a patient’s immune cells inside the body so that they can attack cancer cells or eliminate immune cells responsible for damaging healthy tissue. Unlike conventional ex vivo CAR-T treatments, in which immune cells are removed from a patient’s blood, genetically modified in a laboratory and then returned to the body, in vivo approaches seek to carry out the engineering process within the patient.
The approach could make CAR-T treatment cheaper and easier to develop and administer. Conventional CAR-T therapies are already used against certain blood cancers, but their manufacturing process can be complex and expensive.
An analysis published last month by John Wu of Boston Consulting Group and colleagues found that most of the foundational preclinical research into in vivo CAR-T therapies has been conducted by research groups in the United States and Germany. China, however, has moved particularly quickly towards human testing.
Of 140 in vivo CAR-T drugs being tested in animals and people worldwide, 82% are being developed by groups in China, according to the analysis cited by Nature.
Researchers attribute China’s rapid progress to several factors, including government investment, collaboration between universities, hospitals and companies, a large potential patient population and clinical pathways that allow experimental treatments to reach human trials relatively quickly.
Emily Blyth, a physician and cellular-therapy researcher at the Westmead Institute for Medical Research in Sydney, Australia, said China’s progress had been building for at least five years and involved a large volume of work supported by the government. She added that international groups were increasingly interested in collaborating with Chinese researchers.
Early clinical findings have attracted further attention. The first human trial of an in vivo CAR-T treatment for an autoimmune disease reported results in September 2025. Five people with treatment-resistant lupus experienced improvements in their symptoms three months after treatment, with no serious side effects reported in the trial.
Earlier in September 2026, Chinese researchers reported improvements among 16 people with autoimmune diseases who received in vivo CAR-T therapy. The results add to growing interest in using the technology beyond cancer, although the small number of patients means that substantially larger studies will be needed to establish its safety and effectiveness.
The technology is also beginning to attract attention outside China. The US Food and Drug Administration approved the first China-developed in vivo CAR-T therapy for clinical testing in the United States in September 2026. The treatment is being developed for blood cancers.
Xiaoyuan Chen, a chemist at Shandong Cancer Hospital and Institute in Jinan, who is developing an in vivo CAR-T treatment for cancer and fibrosis, said successful development in China could accelerate innovation, lower treatment costs and generate clinical evidence that could benefit patients internationally.
Researchers caution, however, that the field remains at an early stage. Developers will need to demonstrate that the therapies provide durable benefits and determine whether they can avoid or control serious toxicities associated with existing ex vivo CAR-T treatments before widespread regulatory approval becomes possible.
One important factor behind China’s position is its system of investigator-initiated trials, or IITs. According to Wu and colleagues, the pathway has played a major role in the country’s rapid expansion of cell and gene therapies. Researchers can use IITs to test experimental treatments in patients and generate early clinical evidence without the same direct involvement from the national drug regulator required for conventional drug-development pathways.
The country’s large trial population can also reduce development costs. Wu estimates that conducting comparable trials in China may cost roughly half as much as running them in the United States.
China introduced new IIT regulations in May covering emerging biotechnology treatments, including cell therapies. The rules allow hospitals, in certain circumstances, to sell effective treatments directly to patients with rare diseases without conventional regulatory approval.
At the same time, the regulatory changes have created new requirements for experimental CAR-T research. At least six in vivo CAR-T trials have been paused or withdrawn since May while developers address regulatory requirements.
Under the revised system, only leading research and teaching hospitals, known as sanjia hospitals, can conduct IITs. China’s National Health Commission can also require additional assessments and can order trials to be suspended, modified or cancelled when ethical concerns arise. Trials involving international partners or companies require additional approvals.
Chen said the restructuring should be viewed as an effort to improve safety rather than as a policy designed to restrict development. With in vivo CAR-T therapies still in their early stages, researchers face the challenge of proving that their potential advantages can be achieved without introducing unacceptable risks.
China’s lead in human trials therefore reflects not only the country’s research capacity but also the speed with which experimental cell therapies can move from laboratories into clinical testing. Whether that advantage translates into widely approved treatments will depend on the results of larger trials and the ability of developers to establish long-term safety and efficacy.
