China Dominates Global In Vivo CAR-T Therapy Development

China has emerged as a hub in developing in vivo chimeric antigen receptor T-cell, or CAR-T, therapies, accounting for 82% of the 140 such treatments currently undergoing animal testing or clinical trials worldwide, according to a study published on August 28 in Nature Reviews Drug Discovery.

The analysis, led by Boston Consulting Group managing director Jon Wu and his research team, highlights a significant shift in how advanced cell and gene therapies move from the laboratory into human testing. Traditional CAR-T treatments, which have transformed care for certain types of leukemia and lymphoma, rely on an ex vivo approach. In these conventional therapies, physicians extract a patient’s T cells, alter their genetics in a laboratory to target specific markers, multiply them, and infuse them back into the patient.

While effective, the ex vivo process requires separate cell collection, custom laboratory cultivation, and complex manufacturing, making it time-consuming and expensive. By contrast, in vivo CAR-T therapies use gene delivery vehicles administered directly into the patient’s body to transform T cells internally. This approach eliminates the need for separate cell harvesting and external incubation, potentially simplifying production and lowering costs.

Global Development Trends and Clinical Trial Acceleration

Although basic and preclinical research in the in vivo CAR-T field has been spearheaded primarily by researchers in the United States and Germany, Chinese research teams and companies are moving into clinical trials at a much faster pace. Wu’s research team found that among the 140 in vivo CAR-T therapies in development globally, 82% are being advanced by Chinese developers.

Clinical milestones are following this development volume. On September 2, a research team from Tongji Hospital at Huazhong University of Science and Technology in Wuhan published results in the New England Journal of Medicine detailing the treatment of 16 autoimmune disease patients using an in vivo CAR-T therapy, which demonstrated symptom improvement in some participants. International expansion has begun, with the U.S. Food and Drug Administration approving clinical trials for a China-developed in vivo CAR-T therapy for blood cancer this month.

Institutional Support and the Investigator-Initiated Trial Framework

Experts attribute China’s rapid ascent in in vivo CAR-T development to sustained government backing, close collaboration among research institutions, hospitals, and commercial enterprises, and access to large patient populations. Emily Blyth, an associate professor at the Westmead Institute for Medical Research in Australia, explained in an interview with Nature that research related to in vivo CAR-T has accumulated rapidly in China for at least five years, with a significant portion receiving government support.

Xiaoyuan Chen, director of the Center for Nuclear Medicine and Molecular Imaging at Shandong Cancer Hospital in China, noted that the country has a high volume of autoimmune disease patients and a substantial population with limited treatment alternatives, many of whom are willing to participate in clinical trials.

The study authors highlighted the Investigator-Initiated Trial, or IIT, system as a primary driver of this growth. The IIT framework allows hospitals and researchers to test new technologies on patients without requiring formal clinical trial authorization from national drug regulators, provided the studies receive institutional ethics committee approval and operate under the supervision of the National Health Commission. Wu’s research team estimates that utilizing large clinical trial cohorts and the IIT system cuts clinical trial costs in China to roughly half of what identical trials would cost in the United States.

Regulatory Shifts and Emerging Hurdles

Despite rapid growth, Chinese regulators are tightening oversight of cell and gene therapies. Under new regulations introduced in May, IITs can now only be conducted at ‘Grade A tertiary hospitals’, which represent the top tier of China’s healthcare system.

The National Health Commission retains the authority to demand secondary evaluations, halt, modify, or cancel clinical trials if ethical concerns arise, and require additional approvals for trials involving overseas institutions or companies. Following the implementation of these regulatory changes, at least six in vivo CAR-T clinical trials were suspended or withdrawn after failing to meet the updated requirements.

At the same time, the updated framework includes provisions allowing certain new technologies targeting rare diseases to provide treatment and charge patients before receiving formal drug approval, provided clinical trials demonstrate efficacy.