Second child death reported in Chinese CRISPR trial for Duchenne muscular dystrophy raises safety concerns

A second child death has been reported from a Chinese CRISPR gene-editing trial aimed at treating Duchenne muscular dystrophy, reviving concerns about the safety and transparency of such studies.

The trial is being run by HuidaGene, a Shanghai-based startup that presented early data at the American Society for Gene and Cell Therapy meeting last year. The company used a regulatory pathway that permits Chinese hospitals to start studies without direct oversight from national regulators.

Initial results from the first two patients were modest and did not clearly demonstrate therapeutic benefit. The CEO said a higher dose would be tested, but the firm stopped providing updates for more than a year.

The recent death has prompted experts to criticize the lack of public information and the looser oversight model, arguing that stronger regulatory review is needed before gene-editing therapies move forward.