Xinhua Hospital in Shanghai, China, hosted one of the gene-therapy trials linked to a child’s death last year.
Credit: Costfoto/Sipa USA/Alamy
Researchers, ethicists and legal scholars say the deaths of two children in separate gene-editing clinical trials in China raise serious concerns about research oversight, informed consent and transparency. The cases could also have far-reaching consequences for China’s rapidly expanding biomedical industry.
On 5 August, Shanghai-based gene-editing company HuidaGene Therapeutics announced that a boy had died during a clinical trial conducted in 2025. An investigation by Science and Retraction Watch follows the disclosure, in March last year, that a six-year-old girl had died after receiving another experimental gene therapy.
The girl’s trial was led by neuroscientist Zilong Qiu of Shanghai Jiao Tong University and clinician Yongguo Yu of Shanghai’s Xinhua Hospital. On 26 July, the university published a study about the trial and related preclinical research in a journal. Nature1 The university did not respond to questions about its investigation.
Gene therapy can cause serious side effects, including severe immune reactions that can be fatal.2 At least a dozen people have died in clinical trials of gene therapies in the United States, Europe and Russia over the past three decades.
Researchers say the two Chinese trials involved potential ethical violations and lacked transparency. “This will do, or has done, significant damage to the credibility of Chinese institutions,” said Joy Zhang, a sociologist of science at the University of Kent in Canterbury, England.
The cases have also focused attention on China’s fast-growing investigator-initiated trials, or IITs. These studies allow researchers to test treatments rapidly, often without direct oversight from the country’s drug regulator. In May, however, the Chinese government tightened its rules for such trials.
Jiayou Shi, a lawyer at the National Center for Civil and Commercial Law at Renmin University of China in Beijing, said the deaths could lead to stricter regulations for gene-therapy research. Zhang said the government might suspend some gene-editing research or impose tougher requirements on funding and clinical oversight.
Chinese authorities have responded forcefully to similar incidents in the past. After biophysicist He Jiankui breached research ethics and Chinese regulations by creating gene-edited babies in 2018, the government strengthened laws governing human gene editing and sentenced him to prison. Zhang said, however, that He is widely viewed as a “rogue scientist”, whereas Qiu is an established researcher and influential science communicator.
Ethical concerns surrounding the gene-therapy trial
According to Science and Retraction Watch, pseudonyms were used for the girl and her parents in accounts of Qiu’s trial. The child had a mutation in the CHD3 gene, which causes Snyder-Robinson syndrome, a rare condition associated with physical differences and impaired intellectual development. Qiu and his colleagues used gene editing to correct the mutation. The treatment was delivered using an adeno-associated virus (AAV), which was injected into the spine and transported to the brain.
Fyodor Urnov, a researcher at the University of California, Berkeley, who studies molecular therapies including gene-editing treatments, said the research and medical teams made “terrible mistakes”. One concern was that Qiu reportedly communicated directly with the family, creating a potential conflict of interest because he had developed the treatment. Qiu did not respond to questions about the issue.
Researchers also reportedly received toxicology results showing that all four monkeys tested had suffered liver damage after receiving the treatment. The girl was treated at Xinhua Hospital one month after the team received evidence of that damage. Urnov said the human trial should not have proceeded if the preclinical results showed that the therapy had harmed the animals.
Neither Qiu nor Yu responded to requests for comment on the toxicology findings.
Philippe Campeau, a medical geneticist at the University of Montreal in Canada who helped characterize Snyder-Robinson syndrome, said the girl’s symptoms were mild. He questioned the decision to make her the first patient to receive a potentially dangerous experimental treatment.
The girl’s death has been compared with that of Jesse Gelsinger, a US teenager who became the first person to die in a gene-therapy clinical trial, in 1999.3 Hank Greely, a legal scholar at Stanford University in California who studies the ethics of emerging biotechnology, said Gelsinger had a metabolic disorder that prevented his body from removing ammonia. Like the Chinese girl, however, his underlying condition was not immediately life-threatening.
Gelsinger’s death continues to influence safety standards for gene-therapy trials, Urnov said. The US Food and Drug Administration barred the principal investigator at the University of Pennsylvania in Philadelphia from conducting research, while the university halted a major clinical trial for five years and reached a settlement with the Gelsinger family. The FDA also introduced new rules to strengthen protections for participants in gene-therapy studies.
“The field has come a long way since that tragedy, but that doesn’t mean the field is perfect,” Urnov said.
Another concern is that Qiu co-authored a preclinical study reporting that the gene therapy worked in mice with the same genetic defect, but did not disclose that the treatment had been tested in a girl who later died. The preclinical paper was submitted in December 2024, before the trial, but was not accepted for publication until January, five months after the girl’s death. Campeau reviewed the study and said that it did not mention that the therapy had been administered to a human participant who had died.
On 29 July, Nature issued an editor’s note stating that concerns had been raised about the article and that an investigation was under way. Nature’s news team is editorially independent from the team that publishes research manuscripts and from its publisher, Springer Nature.
Following the editor’s note, Deputy Editor Victoria Aranda said that no information indicating that human trials were planned or under way had been shared with the journal. She added that no data related to human treatment had been included or mentioned in any version of the submitted study.
“We would like to emphasize that we take the concerns very seriously and are committed to acting as soon as possible while ensuring that a robust and comprehensive investigation is conducted,” Aranda said.
Qiu did not respond to requests for comment on the trial or the research paper.
Second child dies in separate gene-editing trial
HuidaGene’s clinical trial involved a treatment for Duchenne muscular dystrophy (DMD), a progressive and ultimately fatal genetic disease. The company used gene editing to alter the faulty gene responsible for the condition. As in Qiu’s trial, the treatment was delivered using an AAV vector.
Four children took part in the trial. According to HuidaGene, the boy who died was the last participant to enrol. He died from acute respiratory distress syndrome, which the company described as a side effect of the treatment.
Lizzie Li, who studies China’s economic and technology policy at the Asia Society Policy Institute in New York City, said the ethical justification for testing a high-risk treatment was stronger in this case because DMD is fatal.
Shi said, however, that the HuidaGene trial also raised concerns. The boy who died received a high dose of the AAV-based treatment, and high doses of AAV vectors have been linked to severe complications and deaths in other gene-therapy trials.4,5,6
Fangxin Li, HuidaGene’s general manager for China, said that the safety of AAV-based treatments cannot be assessed by dose alone. Other factors include the viral vector, the disease being treated and the route of administration.
Li said the dose was similar to those used in two other paediatric DMD gene-therapy trials and in an approved gene therapy for spinal muscular atrophy. The boy received the treatment only after safety data from the lower dose had been reviewed by the trial’s safety board and after information from another participant who had received the higher dose had been considered.
According to Li, neither the participant who first received the high dose nor the two boys who received the lower dose developed the same symptoms. All three remain under observation.
Source: www.nature.com