Child’s death spotlights clinical trial concerns in China

The death of a six-year-old girl who was taking part in a clinical trial has raised troubling questions for scientists in China about ethical oversight and transparency in biomedical research.

The child died in March 2025 after receiving gene-editing therapy in Shanghai but the incident was never reported publicly, despite accounts of the high-profile experiment being featured in prestigious journals.

An investigation by Science and Retraction Watch has revealed that the girl received gene-editing therapy to correct a genetic mutation in her brain impacting her cognitive development.

The clinical trial was conducted at Shanghai Xinhua Hospital, which is affiliated with the Shanghai Jiao Tong University School of Medicine. The six-year-old girl was the first person in the world to receive a gene-editing therapy designed to target the brain.

The experimental treatment, developed by prominent neuroscientist Zilong Qiu of the university’s Songjiang Research Institute, involved infusing trillions of viruses into the fluid at the base of the girl’s spine. But the child had a severe reaction to the treatment and died within the week.

Her parents, who showed accounts and records to Science and Retraction Watch, wished for their daughter and family to retain anonymous. According to the investigation, a report issued by the hospital’s ethics board concluded her death was “definitely related” to the infusion treatment.

The Chinese team that had treated the girl published a paper related to the experimental therapy in Nature earlier this year, and it was received favourably at the time. But it contained no mention of the girl’s death, her family’s involvement, nor problems reported in earlier animal trials.

The paper also omitted details that the girl’s parents had made financial contributions of more than $800,000 (£600,440) to fund the research for the development of the gene-editing therapy.

The parents have asked the authors to withdraw the paper after their daughter’s death, a sentiment shared by several scientists interviewed by Science and Retraction Watch who have called for transparency and accountability.

Steven Gray, professor in paediatrics at the University of Texas Southwestern Medical Center, told the investigation, “This shouldn’t have gone to trial”. 

As China continues to build a reputation for world-class biomedical research, the case has cast further doubt on whether there is sufficient regulation and safeguarding in the nation’s clinical trials and hospital research.

According to the investigation, no action has been taken against Qiu, the Nature paper has not been retracted and the girl’s parents have not received any compensation. 

rosalind.skillen@timeshighereducation.com