A child has died in a gene-editing clinical trial in China, and the death was disclosed only after a months-long investigation by STAT, rekindling debate about safety and transparency in investigator-led trials run under the country's looser oversight pathway.

The case involves Shanghai-based HuidaGene, which tested CRISPR gene editing in children with Duchenne muscular dystrophy — one of the world's first trials of its kind. At a major US gene therapy conference in 2025, the company's CEO presented early data from two patients and said it would test a higher dose. Then the company went silent for 15 months: its CEO and chief technology officer quietly departed, and in February a clinical trial registry updated the study's status to 'complete.'

After repeated questions from STAT, HuidaGene issued an update on Wednesday confirming the child's death. The study ran under a regulatory pathway that lets Chinese hospitals start trials without approval from national regulators — the same kind of framework implicated in a previously reported case, in which a six-year-old girl died after receiving the world's first brain-directed gene-editing therapy at Shanghai's Xinhua Hospital, an outcome Science and Retraction Watch revealed in July.

Experts quoted by STAT said the delayed disclosure highlights a 'lack of transparency' that could erode trust in a field China is racing to lead.

Sources