The death of a minor during a clinical trial involving an experimental gene-editing therapy has prompted one of China’s leading medical universities to launch an investigation. The case has heightened concerns over how biomedical research is supervised and ethical standards are upheld in the country.
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Shanghai Jiao Tong University School of Medicine announced the establishment of a task force that will conduct a comprehensive review of the clinical trial carried out last year at Xinhua Hospital, along with a scientific study linked to the case.
The decision to investigate came several days after the publication of a report by the U.S. journal Science and the Retraction Watch platform. According to the report, the clinical trial ended unsuccessfully, and development of the treatment was partially funded by the girl’s parents, who say they paid approximately $860,000.
The family says doctors did not inform them that the therapy could have fatal consequences. The parents had hoped the intervention would correct the genetic defect responsible for their daughter’s developmental delays.
The girl had been diagnosed with the rare Snijders Blok-Campeau syndrome, a genetic disorder that causes neurological and developmental problems. Seeking treatment, the family approached renowned neuroscientist Qiu Zilong, who had developed a personalized therapy using the advanced gene-editing technology known as “base editing.”
The report notes that she would become the first person in the world to receive this type of therapy directly in the brain. Before it was administered to the girl, experiments had been conducted on mice and monkeys, although several scientists have raised doubts about the reliability of the preliminary data.
A few days after receiving the treatment, the girl developed a high fever and kidney damage. She was transferred to intensive care but did not survive.
A review by the hospital’s ethics committee found that the death was “clearly linked” to the experimental therapy. Despite this conclusion, disciplinary penalties for those responsible were limited. The doctors and institutions involved did not make the case public, while the results of a related study were published in the prestigious journal Nature.
In September 2025, Xinhua Hospital was fined approximately $3,600 by local health authorities. The penalty was imposed because the clinical trial had not been properly registered and the institution had failed to ensure the necessary oversight. One of the doctors responsible received only a verbal warning.
Only a few months later, the parents discovered that Nature had published a study by the research team on correcting the genetic mutation that caused their daughter’s illness. The article mentioned neither the human trial nor her death. The family requested that the publication be retracted and the case investigated, but the university rejected the request.
Nature said it had been unaware that a human clinical trial had been conducted and announced that it was reviewing the concerns raised. The journal’s editors also said they were shocked by the news of the child’s death and the circumstances under which the treatment had been carried out.
The case has reignited debate over the ethics of genetic research in China, years after the 2018 scandal. At the time, scientist He Jiankui announced the birth of the first babies with genetically modified DNA. Chinese authorities subsequently pledged to strengthen the regulatory framework, but experts say the latest case shows that serious problems with transparency, oversight and accountability persist.
In May, Beijing approved new rules for advanced biomedical technologies. The measures call for stricter oversight of clinical trials and mandatory reporting of adverse effects. However, according to experts, the main challenge remains maintaining a balance between rapid scientific progress and ensuring patient safety.
