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An experimental gene therapy trial targeting the brain in Shanghai has resulted in the death of a six-year-old girl, according to an investigation published by the magazine Science and the scientific watchdog site Retraction Watch. The case has prompted the Shanghai Jiao Tong University School of Medicine to open a formal inquiry into the March 2025 incident at Xinhua Hospital, raising urgent questions about oversight and ethics in China’s rapidly expanding biomedical sector.
First Human Subject Receives Base Editing for Rare Disorder
The Shanghai Jiao Tong University School of Medicine announced the formation of a working group to conduct an exhaustive investigation into the death. According to Science, the six-year-old child—referred to as "Mei"—was the first person in the world to receive a brain-targeted gene-editing therapy.
The treatment utilized a technique known as base editing, designed to correct a specific genetic anomaly causing the Snijders Blok-Campeau syndrome, a rare developmental disorder affecting fewer than 300 patients globally. Prior to human trials, lead researcher Qiu Zilong and his team successfully replicated the mutation and the syndrome in mice and monkeys, leading the Xinhua Hospital ethics committee to approve the clinical trial in December 2024.
High Fever, Organ Failure, and Omitted Monkey Safety Risks
One week following the injection, Mei developed a high fever and acute kidney injury before being rushed to intensive care, where she died. According to Xinhua Hospital’s ethics committee, the child’s death was directly linked to the treatment.
Documents and recordings reviewed by Science revealed that preclinical trials on monkeys showed significant liver damage in multiple primates and kidney damage in at least one animal. The family stated that medical personnel assured them there would be no safety catastrophes, failing to properly disclose these severe risks. Yanzhong Huang, a senior fellow for global health at the Council on Foreign Relations, told CNN that the case exposes systemic vulnerabilities in China’s regulatory landscape regarding safety and transparency.
The incident occurs against the backdrop of an unprecedented boom in Chinese biotechnology. According to a 2025 analysis by Chinese researchers cited by CNN, clinical trials in China have surged since 2015, encompassing more participants across numerous trials—double the volume of trials supervised by national drug regulators during comparable periods. Data from the Cure Innovation Index indicates that China’s share of global clinical trial starts rose from 1% in 2009 to 32% in 2025, closely approaching the United States at 35%.
Despite this rapid expansion, regulatory enforcement has faced scrutiny. In September 2025, health authorities in northern Shanghai fined Xinhua Hospital approximately $3,600 for failing to properly supervise the clinical trial and omitting mandatory registration in the national private-sector research database, as reported by Science. Additional reports indicated that the child’s father provided gifts, including electronics and liquor, to researcher Qiu Zilong, though investigators found no formal solicitation by the doctor.
Rejected Retraction Demands and New Beijing Oversight Rules
In May 2025, Beijing implemented new regulations governing advanced biomedical technologies, centralizing oversight and mandating strict reporting of adverse events for researcher-initiated clinical trials.
Mei’s parents subsequently requested the retraction of the original research paper published in Nature, which detailed the preclinical feasibility of correcting the genetic mutation without mentioning the subsequent fatality. The university rejected the request. On July 26, 2025, the medical school issued a public statement confirming it is taking the online reports regarding the clinical studies conducted at Xinhua Hospital with utmost seriousness.
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