Chinese Biotech HuidaGene Discloses Second Child Death in Investigator-Led CRISPR Trial

Shanghai-based HuidaGene Therapeutics disclosed on August 5, 2026 that a participant in its HG302 trial — testing a proprietary CRISPR variant (hfCas12Max) delivered intravenously via AAV vector to restore dystrophin production — died in August 2025 after receiving a high dose of the therapy for Duchenne muscular dystrophy. The company attributed the death to acute respiratory distress syndrome arising from a severe immune reaction to the AAV vector. The investigator-initiated trial, run at Shanghai Children's Medical Center under a Chinese regulatory pathway that allows hospital-based studies without government oversight, enrolled four boys aged 4–8; the fourth patient, who died, had received the highest viral dose tested. HuidaGene disclosed the death only after a months-long STAT News investigation, roughly a year after it occurred and without an independent data safety monitoring board reviewing the dose-escalation decision that preceded it — reigniting debate over transparency and safety oversight of investigator-led gene-editing trials in China, following a similar earlier case.