On Wednesday, August 5, according to the U.S. health news website STAT, a Chinese boy suffering from Duchenne muscular dystrophy (DMD) died after participating in an early clinical trial initiated by HuidaGene in Shanghai. The clinical trial registration for the project indicated that the deceased boy was between 4 and 8 years old.
HuidaGene simultaneously released a Chinese statement stating that the incident occurred in August 2025. The participant experienced acute respiratory distress syndrome (ARDS) due to severe complement and cytokine activation after receiving high-dose adeno-associated virus (AAV) vector systemic administration.
This is the second case of child death caused by Chinese gene editing trials revealed in international journals within two weeks. Additionally, it is a medical event in China that was reported by foreign media a year after the patient’s death.
Science journal disclosed the failed gene editing trial led by the Shanghai team of Qiu Zilong on July 23, involving the death of a 6-year-old developmentally delayed girl after receiving brain-targeted gene editing.
STAT pointed out that these two cases share striking similarities: both trials were initiated by researchers with weak oversight by authorities, patients died from fatal immune reactions triggered by viral vectors, and initially, the parties involved remained silent while the research continued. It is reported that STAT has conducted an extensive investigation into HuidaGene for several months and has repeatedly sought the company’s response to the incident.
HuidaGene stated in its announcement that the deceased child was the last participant in the established protocol, and the other three participants did not exhibit similar severe clinical symptoms and are still under long-term follow-up.
The complete results were submitted for peer review in January 2026, with more scientific details to be made public after the publication of the paper.
According to the information left by HuidaGene in the ClinicalTrials.gov registry, the clinical trial started in November 2024, with the planned inclusion of male children aged 4-8. The experiment was originally scheduled to complete the main part in August 2026 and data analysis by June 2027. As of August 3, 2026, HuidaGene made the final update on the relevant experiment records, stating that it is ongoing but no longer recruiting patients.
ClinicalTrials.gov is one of the most internationally influential clinical trial registration agencies used to track and report trial results.
Based on a Chinese news release by HuidaGene in December 2024 about the first subject receiving treatment, the experiment involved their newly developed DNA editing therapy HG302, using adeno-associated virus to deliver the CRISPR editing tool into the body. According to preclinical studies, the company believed that the required dosage of their therapy was lower than some existing AAV therapies to reduce immune-related risks.
STAT reported that HuidaGene’s CEO, Lu Yingming, publicly disclosed at the American Society of Gene and Cell Therapy (ASGCT) annual meeting in May 2025 that only trace amounts of dystrophin, the key protein missing in DMD patients, were detected from…
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The departure of Lu Yingming and Chief Technology Officer TJ Cradick had reportedly no connection with the death incident. They were recruited by the company in hopes of establishing a subsidiary in the United States to commercialize their projects and tap into more lucrative markets.
Li Fangxin, the general manager of HuidaGene, mentioned in May that the company had made adjustments, ceasing large-scale virus systemic infusion clinical trials like for DMD. Instead, the focus would be on injecting small amounts of virus directly into the brain or eyes.
The consecutive exposure of two gene editing death incidents at Shanghai hospitals within two weeks may intensify external skepticism on China’s intentionally loose regulatory pathway for Investigator-Initiated Trials (IIT) to conduct clinical trials.
The IIT independent trials in China are currently in a state of disarray. Until May 2026, the National Medical Products Administration of China issued a document #818, stipulating that only top-tier hospitals can conduct independent trials. Hospitals must complete non-clinical safety and efficacy assessments and obtain approval from the institutional ethics committee.
According to the case of the 6-year-old girl disclosed by Science journal, such ethics committees appeared virtually useless, even allowing trials to proceed before animal experiment results were out.
Kiran Musunuru, a gene editing researcher at the University of Pennsylvania, expressed his concerns about the lack of central supervision and transparency in China’s IIT research to STAT. He mentioned that based on HuidaGene’s experiment records, the research results should have been published in preprint form months ago but remain unheard of.
“The company should have been transparent about this,” he said. “But now there is still no information.”
Both Lu Yingming and Cradick told STAT that their departure from HuidaGene was unrelated to the death incident. The company recruited them to establish a subsidiary in the U.S. for the commercialization of their projects to explore more profitable markets.
“The last time I left was in August (2025),” Cradick told STAT. “I wasn’t involved when they started treatment or planned higher doses.”
HuidaGene, founded by Yang Hui, a researcher at the Institute of Neuroscience, Chinese Academy of Sciences, in 2018, focuses on designing, modifying, and developing new CRISPR gene editing tools and innovative gene therapies. In the same year, Yang Hui was accused of plagiarizing a research report from visiting Professor Fu Xiangdong.
Within just 7 years, leveraging Investigator-Initiated Trials (IIT) fast track initiated by researchers, HuidaGene has advanced six gene editing therapies to human trial stages.
Gene therapy trial-related severe adverse events are not uncommon. Over the past eight years, at least a dozen patients have died during clinical trials or after approved gene therapies. However, cases like those in China where the truth needs to be exposed by foreign media after months or years before reaching Chinese audiences are quite rare.