Renzong Qiu tells the story of the death of a six-year-old girl, a research participant in a base-editing gene therapy trial in China, and argues that research participants’ interests should never be overridden by scientific or social interests.

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In March 2025, a six-year-old girl—referred to by the pseudonym “M”— underwent an experimental brain-targeted base-editing trial led by neuroscientist Qiu (given name: Zilong), a physician at Xinhua Hospital, Shanghai Jiaotong University. Her experimental therapy involved a spinal injection of trillions of modified adeno-associated viruses carrying a custom base editor; these viruses were designed to migrate into and penetrate brain neurons to correct a specific genetic mutation. This mutation—a substitution of a T base for a C base—was responsible for her neurodevelopmental disorder, known as Snyder–Robinson–Kampho syndrome. In a few days after the injection, M developed a high fever and signs of acute kidney injury. She passed away on March 31 after the high viral load triggered a catastrophic immune response.

This was not an ordinary clinical trial. The investigator used a novel base editing technology to repair a wrong base which is caused by the mutation of a gene in M’s neural cells. Base editors are themselves macromolecular complexes—typically formed by fusing a deactivated Cas protein with a deaminase—that require a delivery vehicle, such as a modified adenovirus, to cross the cell membrane and reach the DNA inside. In other words, the intervention for M involved injecting her with viruses in order to “fix” the DNA in her brain.

Photo Credit: Victoria Borodinova/publicdomainpictures.net. Image Description: A computer generated image of a DNA.

Base editing technology itself is far from mature. The field has only three years of experience in clinical trials. Base editing carries risks such as off-target effects, unintended modification of adjacent bases, accidental DNA damage, and immune reactions—potentially even resulting in death. The potential safety issues remain uncertain or unknown, necessitating extreme caution when conducting clinical trials involving base editing.

This was an investigator-initiated trial —a type of study not widely understood by the Chinese public, and a regulatory pathway with little oversight. These are clinical trials conceived, designed, and led by researchers or clinicians. They can be initiated without regulatory approval by China’s National Medical Products Administration. In the case at hand, the funding for the study involving M was provided by the patients’ parents and channeled into the lead researcher’s own company.

Zilong Qiu appears to be a second He Jiankui: a rogue scientist lacking integrity.

Clinical trials should be ethically prohibited for new therapies that pose risks of severe, irreversible harm (such as disability) or death, as the risk-benefit ratio in such cases is inherently unfavorable. Trials carrying a risk of death are not permitted for six-year-old children with mild symptoms. Successful clinical trials of base editing to date consist of only a handful of cases; with such small sample sizes and a lack of control groups. As a senior scientist, Qiu ought to have been aware of this.

Furthermore, based on the information currently available, Qiu completely failed to meet the requirements for valid informed consent. He presented only the positive aspects while omitting the negatives, informing the parents merely that the base editor had successfully functioned in the brains of mice and monkeys with minimal risk. He did not disclose risks such as immune reactions which may cause death in severe cases. He may have deliberately concealed from the parents the fact that liver damage had occurred in primate studies and kidney damage in mouse experiments.

Participants in clinical trials should also not be required to pay costs. Qiu instructed the parents to transfer funds to his company and the companies owned by his colleagues but failed to disclose that his operation of the company created a conflict of interest.

The study involving M was reviewed by an ethics review committee, but the committee looks to have functioned as a rubber stamp. The committee seems to have not reviewed the results of Qiu’s animal experiments and did not discuss the health implications of the liver and kidney damage observed in the animals. Because of the unfavourable risk-benefit ratio, the committee should have rejected the protocol outright.

This grave incident reveals a lack of even the most basic oversight regarding human clinical trials at the hospital, medical school, and university in question. Relevant regulations issued by multiple government agencies have essentially not been implemented at these institutions. In particular, two pillars of research participant protection—valid informed consent and independent ethical review—have ceased to exist there.

Regulatory oversight must be strengthened for emerging biomedical technologies that involve critical parts of the human body—such as gene therapy, gene editing, invasive brain-computer interfaces, and xenotransplantation. There is reason to believe that the M incident is not an isolated case; similar egregious incidents may be occurring at other institutions but remain tightly concealed from public view. The National Health Commission should conduct a comprehensive review of all clinical trials or research projects of emerging biomedical technologies and involving human subjects.

Scientists and physicians whose work concerns human health should also be prohibited from owning companies related to medical care and human health, so as to effectively avoid conflicts of interest and prevent serious harms. To prevent further cases like the death of M, there should be a transparent mechanism to ensure that regulatory bodies and the public are promptly informed of the findings of research projects.

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Renzong Qiu is a scholar at the Institute of Bioethics, Center for Ethics and Moral Studies, The People’s University of China.