Beijing —
China’s top medical university is investigating the death of a young girl from a gene-editing experiment conducted by a leading expert at an affiliated hospital, raising serious concerns about ethics in China’s burgeoning biomedical industry.
Shanghai Jiao Tong University Medical School announced that it has set up a working group to “conduct a comprehensive investigation” into clinical trials conducted last year at Xinhua Hospital and related scientific papers.
The announcement came days after the US-based journal Science and Retraction Watch, a scientific accountability publication, released a joint report claiming the girl’s parents paid $860,000 to develop the treatment and detailing the failure of a partially funded clinical trial.
The girl’s parents told the media that doctors did not warn them that their daughter could die, and that they were hopeful that treatment would correct the genetic defect behind her developmental disorder, and that they feared it would affect her later life.
The hospital’s ethics committee concluded that the girl’s death was “clearly related” to the treatment, but the penalty was light, and although the results of a related study were published in the prestigious scientific journal Nature, the results were never made public by the doctors or their institutions, the Science report revealed.
The current high-profile case has sparked anger and concern in China, particularly after tightening regulations following another scandal: the 2018 revelation that babies were born from gene-edited embryos by Chinese scientist He Jiankui.
The landscape of modern clinical trials is very different. Gene editing is an emerging form of gene therapy that aims to edit DNA or RNA sequences to repair or remove defective genes that cause various diseases in children and adults. Unlike gene-edited embryos, these changes are not passed on to future generations.
Still, “this case is much more worrying than[He Jiankui’s case]…because it comes from an established scientist at one of China’s most prestigious universities,” said Joy Zhang, director of the Center for Global Scientific and Epistemic Justice at the University of Kent in the UK.
In contrast, he was a little-known entrepreneurial scientist working at a recently established facility when he became the first and only famous researcher to manipulate human embryos for implantation. The move shocked the world scientific community and he was sentenced to three years in prison.
Since then, “the government has made a series of initiatives and commitments about how it wants to strengthen ethics, but clearly this is not the case,” Chan said.
“Seeing similar ethical deficiencies emerge after so many years suggests that there are still gaps in regulation in terms of transparency, safety and accountability,” said global health expert Yangzhong Huang.
“If you look at this lawsuit, there are a lot of mistakes, loopholes, fraud… It’s very unique in that sense, but I worry that in this environment of China’s biomedical boom, this could potentially be the tip of the iceberg,” said Fan, a senior fellow at the Council on Foreign Relations.
Gene therapy, an umbrella term that includes the development of treatments using gene editing, is the driving force behind China’s rapidly expanding biotechnology sector, with researchers competing with those in the United States and elsewhere to advance medical science.
Experts say such research is being boosted by the country’s so-called dual-track regulatory system, which allows treatments to be tested in humans for the first time through “investigator-initiated trials.”
These small-scale clinical trials typically have less oversight than traditional green-light approval pathways. Similar models exist in other countries and are considered critical to accelerating breakthrough treatments and delivering life-saving treatments, especially for rare diseases where there is little commercial incentive for research. But they can also come with risks.
Against this backdrop, the Shanghai couple who shared their story with Science magazine set out on a journey to seek treatment for their young daughter.
The girl had been diagnosed with a rare genetic defect that causes a neurodevelopmental disorder known as Snyders-Brock-Campeau syndrome, Science magazine reported.
The disease can cause severe physical symptoms as well as intellectual disability. The girl’s symptoms were milder than other girls, but her parents decided to consider a special gene therapy, according to Science.
They contacted Qiu Zilong, a prominent neuroscientist at the Songjiang Institute of Shanghai Jiao Tong University School of Medicine. Qiu was known for her work using cutting-edge technology known as base editing to create custom treatments for children with rare diseases.
According to Science magazine, the girl will become the first person in the world to receive brain-targeted gene editing therapy under a treatment plan he developed for her family. According to Science magazine, which examined recordings made by the family of Chiu, the family received assurances from Chiu that “there would be no safety disaster.”
Preparations included preclinical tests in mice and monkeys, and Cui told the family that this proved they should proceed with clinical trials, Science reported.
The hospital’s ethics committee approved the clinical trial in early 2025, before the final results of one of the preclinical studies in monkeys were published, according to a report in the journal Science. Other results are “less convincing,” according to scientists reviewed in Science.
A few days after the girl was treated in March last year, she developed a high fever and signs of kidney damage, was rushed to the ICU, and died. CNN has not been able to independently verify the account.
The list of clinical trials related to experimental treatments has not been updated since last year. When contacted by CNN by phone, one of the trial’s lead doctors, Yu Yongguo, declined to comment and hung up. Chiu did not respond to an email request for an interview. Repeated calls to the medical school’s public relations department went unanswered.
In September 2025, the Shanghai District Health Bureau fined the hospital about $3,600 for failing to properly oversee the trial and register it in a national database as a commercially sponsored study, according to a report in Science magazine, while Yu was asked to undergo verbal counseling by the hospital, the family said. CNN has also reached out to the hospital for comment.
It was in February of this year that Chiu’s family learned that Nature magazine had published his paper and demanded action from the university. The paper describes research into the feasibility of using gene editing to correct the mutation that caused the daughter’s disorder, but there is no mention of human clinical trials or the daughter’s death.
The couple called for the investigation and the paper to be retracted, telling Science they feared the paper could give other parents false hope. The university rejected the request, Science magazine reported.
Victoria Aranda, Nature’s associate editor, said in a statement to CNN that the journal is investigating concerns raised about the manuscript, is in contact with the researchers, and will “take editorial action as appropriate” following the investigation.
“No information was shared with us that any human trials were planned or underway, and no data related to human treatment was shared or mentioned in any version of the submitted study,” Aranda said. “We were shocked to hear of the recent death of a child in a clinical trial and the circumstances surrounding that trial.”
On July 29, an editor’s note was added to Nature. A Chinese-language press release about its publication has been removed from Xinhua Hospital’s website, CNN has discovered.
The scandal comes as Chinese regulators scramble to keep up with a surge in innovation and investment in advanced treatments.
A 2025 analysis by Chinese researchers found that since 2015, physician-initiated clinical trials for cell and gene therapy products have surged, with more than 30,000 participants enrolled in more than 1,000 trials, twice the number of participants in clinical trials for investigational drugs overseen by the country’s regulatory bodies.
In May, the Chinese government introduced new regulations specific to new biomedical technologies, filling what observers said were gaps in how the development of advanced treatments, including personalized treatments, is managed.
The new rules establish a national regulatory framework to govern such trials, including physician-initiated clinical trials like those conducted at Xinhua Hospital, centralizing oversight and requiring reporting of side effects. It is also believed to create a clearer path to commercializing these treatments.
Associate Professor Calvin Ho from Australia’s Monash Law School said: “It is speculative to say whether this governance would have made a difference[in this case]but one would hope that if safeguards like this had been in place… the institutions themselves could have acted more cautiously.”
Rather, the trial took place at a time when “no clear governance structure was in place,” Ho said, adding that balancing innovation in these areas with patient protection is currently a challenge for governments around the world.
Observers said the incident could lead to further crackdowns and surveillance in China in an expedient response that obstructs science. But Beijing is not only balancing innovation and safety, but also global competition, and may not want to slow down.
According to a recent survey of US industry and academic leaders conducted by Cure Innovation Index, China’s share of global clinical trial starts will increase from 1% in 2009 to 32% by 2025, roughly equal to the US’s 35%.
Chinese institutions have the capacity and are conducting cutting-edge research, Huang said.
“But at the same time we are talking about China’s rise as a biotech superpower, we must not forget the other side of the story: the regulatory and ethical challenges that could pose serious obstacles to Chinese science.”
