Mei never reached the age of seven. The little girl, a pseudonym requested by her parents to protect her identity, entered one of the most prestigious pediatric hospitals in Shanghai in March 2025 believing she was embarking on an adventure. She packed her favorite stuffed animals, play-dough, and a tablet loaded with episodes of Peppa Pig. She told her parents it felt like they were going on vacation. In reality, she was about to become the first person in the world to receive a gene editing therapy targeted at the brain.
Seven days later, she was dead. Her death was never publicly disclosed. It was also not mentioned in a scientific article that months later presented the research as a significant breakthrough in the fight against rare genetic diseases.
The tragedy remained buried until a joint investigation by the journal Science and the specialized portal Retraction Watchreconstructed the case from official documents, hospital records, recordings by the parents, and dozens of interviews. The outcome painted a worrisome picture of the lack of transparency still surrounding some cutting-edge clinical trials in China.
Let's start from the beginning. The girl suffered from Snijders-Blok-Campeau syndrome, an extremely rare genetic disease caused by a mutation in the CHD3 gene, leading to neurological development disorders. Although the disease can cause intellectual disabilities, many patients have a normal life expectancy. For Mei, however, hope came wrapped in a revolutionary promise: to directly correct the genetic error in her brain cells using a sophisticated base editing technique.
The project was led by Qiu Zilong, one of the most renowned Chinese neuroscientists in the field of neurodevelopmental disorders. His research positioned China in the global race to develop personalized gene therapies for children with rare conditions, an area where Beijing aims to challenge the scientific leadership of the United States.
Mei's parents embraced this possibility. For nearly two years, they raised around six million yuan (approximately 755,000 euros) from their savings and relatives to contribute to the development of the treatment, convinced that they were offering their daughter the only chance to improve her quality of life. The therapy had passed animal experiments, and the Xinhua Hospital in Shanghai authorized a clinical trial after its ethical committee's approval.
However, as revealed a few days ago by the investigation from Science and Retraction Watch, the committee never reviewed the final report on primate toxicity, which already showed significant liver and kidney damage associated with the treatment. Despite these warning signs, the trial continued.
On March 24, 2025, doctors injected hundreds of trillions of genetically modified viruses carrying instructions to repair the mutation responsible for Mei's disease into her cerebrospinal fluid. During the first hours, everything seemed to be going as planned. But a few days later, persistent fever, lack of urine production, and a sudden drop in platelets began.
The doctors transferred the girl to the intensive care unit. Before entering, she looked at her mother and whispered a phrase that still haunts the family: "Mom, I want to go home." The next day, she passed away. The Xinhua Hospital's committee later concluded that the death was "definitely related" to the experimental treatment and attributed it to thrombotic microangiopathy, a known complication of some gene therapies.
Far from becoming a case that required a review of the safety of such research, the death disappeared from the official narrative. Months later, Qiu's team published a paper in Nature focusing on the preclinical experiments supporting that gene editing therapy.
The article described the promising results obtained in animals and presented the technology as a decisive step towards clinical application, but made no mention that the treatment had already been administered to a patient or that this first intervention had ended in death.
For months, Mei's parents requested the article to be retracted as they believed it offered an incomplete picture of the research and could lead other families to trust in a treatment whose risks had not been transparently disclosed. However, the publication proceeded and was warmly received within the scientific community. Even Chinese state television presented the advancement as a ray of hope for thousands of families affected by rare genetic diseases, while other parents began contacting the research team interested in subjecting their children to similar therapies.
Now, as confirmed by this newspaper, the School of Medicine at Jiao Tong University has established a new research team to review the entire clinical trial conducted at Xinhua Hospital, as well as the scientific article signed by Qiu. In a statement, the university has stated that it has always advocated for "the integrity and standards of scientific research" and maintains a position of "zero tolerance" towards any study that violates ethics or biomedical regulations.
However, the consequences for those responsible have been minimal so far. After the girl's death, the Xinhua Hospital was fined a mere 24,000 yuan (about 3,000 euros) for deficiencies in overseeing the trial, while the responsible doctor received only an internal reprimand. The family did not receive any financial compensation.
Mei's death has reopened the debate about China's peculiar regulatory system for clinical trials. In recent years, many leading hospitals have been able to launch experimental therapies after their own ethical committees' approval, without the need for prior national regulator authorization. This lack of a filter accelerated biomedical innovation, but also raised doubts about controls.
The case has also sparked criticism among prominent experts in gene therapy and bioethics. Experts consulted by Science believe that the researchers downplayed the risks when explaining the trial to the family, did not give enough weight to the toxicity signals observed in animals, and moved towards the clinical phase without solid evidence that the treatment could offer real benefits.
For China, which aims to become a global biotechnology power, the case is a harsh blow. The Asian giant has tried to strengthen the ethical oversight of emerging medical technologies in recent years following the scandal of He Jiankui, the scientist who created the first genetically modified babies. In fact, Last June, the authorities tightened regulations on cellular and gene therapies, limiting these trials to authorized hospitals and prohibiting charging patients to participate in clinical research. The reforms aimed precisely to prevent situations like Mei's, whose death remained hidden while the scientific work continued to be presented as a success.