HuidaGene discloses a CRISPR trial death twelve months late

HuidaGene Therapeutics disclosed on 5 August 2026 that a participant in the high-dose cohort of its HG302-01 trial died in August 2025 — twelve months before the company said so publicly. HG302 is an investigational gene-editing therapy for Duchenne muscular dystrophy, administered systemically in an adeno-associated virus vector. In the company’s own words, the participant “developed acute respiratory distress syndrome in the setting of severe complement and cytokine activation following high-dose systemic administration of an adeno-associated virus vector”. Four participants were enrolled in the first-in-human, investigator-initiated study; three remain under follow-up.

HuidaGene says it has completed an investigation covering laboratory, immunological and pathological analysis and an autopsy, has informed the regulatory authorities, and submitted the full results to a peer-reviewed journal in January 2026, with further scientific detail to follow publication.

It is the second death of a child in a Chinese gene-editing trial to surface within months. The Standard reports that a six-year-old girl died in March 2025 after a brain-targeted gene-editing treatment.