Second child’s death in China gene-editing trial disclosed after months of silence

The deaths of two children in separate gene-editing clinical trials in China have come to light in rapid succession, intensifying scrutiny over the country's regulatory framework for cutting-edge medical research and the transparency of investigator-led studies.

A Hidden Death Exposed

On July 23, a joint investigation by Science magazine and Retraction Watch revealed that a 6-year-old girl died in March 2025, seven days after receiving an experimental brain-directed gene-editing therapy at Xinhua Hospital in Shanghai. The child, who had Snijders Blok-Campeau syndrome — a rare but non-life-threatening neurodevelopmental disorder — was the sole participant in what researchers called the world's first human test of its kind.chinadaily+2

The trial was led by neuroscientist Zilong Qiu of the Shanghai Jiao Tong University School of Medicine. The girl's parents had paid $860,000 to fund preclinical work for the treatment. She died of thrombotic microangiopathy, a condition in which tiny blood clots damage organs, which an internal ethics review at Xinhua Hospital concluded was "definitely related" to the treatment.retractionwatch+2

The death was never publicly disclosed. In February 2026, Qiu's team published preclinical findings in Nature Springer Nature Group without mentioning the human trial or the fatality. On July 29, Nature added an editorial note flagging concerns about the paper. The trial was marked as terminated on ClinicalTrials.gov on July 30.chinadaily+1

Shanghai Jiao Tong University School of Medicine announced on July 26 that it had established a task force to investigate, stating it "firmly opposes any medical research that violates scientific ethics".chinadaily

A Second Death Emerges

Then on August 5, Shanghai-based HuidaGene Therapeutics disclosed that a patient in its Duchenne muscular dystrophy gene-editing trial died in August 2025 after receiving a high dose of its therapy, HG302. The company attributed the death to acute respiratory distress syndrome caused by severe complement and cytokine activation following systemic administration of an adeno-associated virus vector.bloomberg+1

HuidaGene's CEO Alvin Luk had presented early trial data at the American Society for Gene and Cell Therapy conference in 2025, but the company went silent for 15 months afterward, according to STAT News. Luk and Chief Technology Officer TJ Cradick quietly departed the company last summer. The disclosure came only after months of investigation and repeated questions from journalists.statnews

Regulatory Gaps Under Scrutiny

Both trials used pathways that allowed Chinese hospitals to initiate studies without direct oversight from government regulators. Shi Jiayou, a law professor at Renmin University of China, told The Beijing News that subjecting a child with a non-life-threatening condition to known lethal risks violates foundational principles of research ethics.statnews+1

"The lives and safety of research participants must always take priority over world's firsts, journal publications and academic breakthroughs," Shi said.chinadaily