A 6-year-old girl's death in 2025 after receiving a brain-targeted gene-editing therapy in Shanghai has dominated headlines in recent days.

The full story remained hidden for a year, until a joint investigation by Science and Retraction Watch was published on July 23, bringing it to the attention of the industry and the public.

The investigation revealed that the girl, identified by the pseudonym "Mei," suffered from a rare CHD3 gene mutation syndrome. Her parents paid $860,000 to fund the brain-targeted gene-editing research. However, seven days after receiving an AAV vector infusion, Mei died from thrombotic microangiopathy. The hospital's ethics committee determined that the death was "definitely related" to the experimental treatment.

According to the Science investigation, the research team failed to adequately inform the family of the risk of death. After the final primate toxicology report revealed liver and kidney damage, the relevant data were not submitted to the ethics committee for re-review. In addition, approximately $130,000 in research funds were transferred to a researcher's personal account.

In February 2026, the research team published a preclinical paper in Nature. The final paper removed all references to the girl's family and their financial support, and failed to disclose the girl's death or the liver and kidney damage observed in the primate safety studies.

Drug innovation requires courage, and medical progress depends on bold exploration. However, new drug development must always uphold a profound reverence for life. Every step forward must be subject to ethical calibration, regulatory constraints, and transparent oversight.

This incident has exposed systemic problems—including delayed ethics committee reviews, inadequate informed consent, and failure to publicly disclose adverse events—sounding a wake-up call for the entire industry.

Following widespread media coverage, intense discussions erupted within the Tonacea CGT President WeChat group. Founders of multiple cell and gene therapy companies agreed that the issues exposed by the research team represented a serious departure from the fundamental ethics and scientific principles of drug development.

Many expressed that while cutting-edge biomedical exploration requires a pioneering spirit, all scientific research must be conducted with the utmost reverence for life and a firm commitment to research integrity. Complete ethical oversight, transparent trial management, and full risk disclosure are essential to safeguarding the rights of trial participants and strengthening the public health defense line.

Against this backdrop, Tonacea Cell and Gene Therapy Club swiftly launched the Industry Self-Discipline Initiative on Standardizing Research Conduct and Upholding Scientific Ethics. The initiative was co-launched by Zengjiang Cheng(Vice President of the China Food and Drug Safety Promotion Association, Founder and Chairman of Tonacea)，Yu Zhang（Chairman of Tonacea Cell and Gene Therapy Club, Director and Co-CEO of VCANBIO），Grace Zhou（Vice Chairman of the Tonacea Cell and Gene Therapy Club, Founder, Chairwoman and CEO of ImmVira). It quickly garnered broad support from China's CGT companies. As of 8:00 PM on July 27, a total of 91 founders, CEOs, and presidents from the CGT sector had endorsed the initiative—a powerful demonstration of the industry's collective commitment to upholding research ethics and self-discipline.

China's new drug R&D is at a critical stage of transition—from "following" to "running alongside" and even "leading" globally. The CGT field, as one of the most cutting-edge areas in biomedicine, carries the hopes of countless patients and the expectation that China's biopharmaceutical industry will make a leapfrog advance. Yet it is precisely at such a critical juncture that a clear mind and firm bottom lines are most needed.

Technological innovation can be bold, but it must always operate within the framework of ethics and regulations—this is a fundamental consensus of modern medical civilization and a prerequisite for Chinese innovative drugs to gain global recognition. The long-term prosperity of an industry is never built on a few top-journal papers or "first-ever" achievements, but on society's enduring trust in its rigor and sense of responsibility. Once trust is broken, the cost of rebuilding it will be immeasurable.

The launch of this Self-Discipline Convention is laying the groundwork for the future of China's CGT industry—making compliance a habit, integrity a part of its DNA, and ensuring that Chinese innovative drugs not only move fast but also move steadily and far. As the initiative states: "Let this incident serve as a mirror for us: keep our promises in mind, shoulder our responsibilities."

The full text of the convention is as follows: