Child dies in Chinese gene-editing trial for Duchenne muscular dystrophy, raising safety and transparency concerns

A child with Duchenne muscular dystrophy died in August 2025 during a Chinese gene-editing trial of HuidaGene Therapeutics' HG302 therapy, sparking safety and transparency concerns. The death, caused by a severe immune reaction to a high dose of adeno-associated virus vectors, was only disclosed in August 2026 following a media investigation. The incident, along with another trial fatality, intensifies scrutiny on China's investigator-initiated trial system, which bypasses national drug regulators.

Child death in gene trial

• ▪A child with Duchenne muscular dystrophy died in August 2025 after receiving the HG302 gene-editing therapy developed by Shanghai-based HuidaGene Therapeutics.
• ▪HuidaGene Therapeutics disclosed the August 2025 patient death on August 5, 2026, following a months-long investigation by STAT News.
• ▪Clinical trial records indicate that HuidaGene Therapeutics' study for the HG302 gene-editing therapy only enrolled boys between the ages of four and eight.
• ▪The deceased patient was the fourth and final participant enrolled in HuidaGene Therapeutics' clinical trial for the HG302 gene-editing therapy.

AAV vector immune reaction risks

• ▪The deceased patient in the HuidaGene Therapeutics trial received a high dose of HG302, which uses modified adeno-associated virus vectors to deliver DNA-editing components.
• ▪High-dose administration of adeno-associated virus vectors has previously caused fatal immune reactions in other clinical trials globally, including in boys with Duchenne muscular dystrophy.
• ▪HuidaGene Therapeutics attributed the August 2025 patient death to acute respiratory distress syndrome resulting from a severe immune reaction to the HG302 therapy.

China investigator-initiated trial oversight

• ▪HuidaGene Therapeutics CEO Alvin Luk and Chief Technology Officer TJ Cradick both departed the company in the summer of 2025.
• ▪Chinese authorities and an affiliated Shanghai university launched investigations following the previously unreported death of a young girl in a separate investigator-initiated gene-editing trial.

HuidaGene leadership departures

• ▪Former HuidaGene Therapeutics CEO Alvin Luk presented early trial data from the first two Duchenne muscular dystrophy patients at the American Society for Gene and Cell Therapy convention.
• ▪HuidaGene Therapeutics did not issue any press releases for 15 months following its presentation at the American Society for Gene and Cell Therapy convention.

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