Child's death in China gene therapy trial sparks ethics debate

The death of a six-year-old Chinese girl following an experimental gene therapy for a rare genetic disorder has reignited discussions about the ethics and safety standards in medical research. The incident, which occurred in March 2025 but was not disclosed at the time, involved a child identified pseudonymously as 'Mei,' who had Snyder-Robinson syndrome.

This rare genetic condition affects fewer than 300 people worldwide and results from a mutation impacting a key protein regulating DNA. While it causes developmental and cognitive issues, it is not considered fatal with appropriate medical care. Mei's parents contacted Chinese neuroscientist Qiu Zilong in 2023, who was developing gene therapy for a similar condition and agreed to create a personalized treatment using gene-editing techniques.

The project utilized 'base editing,' a cutting-edge gene-editing method, delivered via a modified viral vector. The family reportedly funded around 756,000 euros for the research through various institutions and companies. Pre-clinical trials on mice showed promising results, improving disease symptoms. Subsequent tests on monkeys indicated the viral vector could reach brain cells, prompting the research team to seek ethics committee approval for human trials.

However, an investigation revealed that the ethics committee at Xinhua Hospital, affiliated with Shanghai Jiao Tong University, was not fully informed about toxicity studies. These studies, conducted by Primed company, showed liver damage in all participating monkeys, with one also experiencing kidney complications. Despite these findings, the committee approved the human trial. Mei received the gene therapy in March 2025 via cerebrospinal fluid injection and died just a week later. The hospital's ethics committee concluded the death was directly linked to the therapy, likely causing micro-clots in blood vessels, a complication observed in one of the monkeys.

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The doctor found that my son's X-ray results were not normal and asked me to go to the hospital for further examination.