Two undisclosed gene-therapy deaths raise doubts about China's fast-track trial pathway

Two recent deaths in China’s early‑stage gene‑therapy trials have drawn international attention. Both patients suffered severe immune reactions after receiving high‑dose viral vector treatments, and the incidents were not disclosed at the time they occurred.

The first case involved a six‑year‑old girl with a rare neurodevelopmental disorder who died last year from a severe immune response shortly after an experimental base‑editing therapy, a story reported by Science and highlighted by Retraction Watch.

The second case concerned a young boy enrolled in an investigator‑initiated trial by HuidaGene Therapeutics for Duchenne muscular dystrophy. He died of acute respiratory distress syndrome after a CRISPR‑based treatment, a finding uncovered by Stat.

These tragedies have sparked scrutiny of China’s investigator‑initiated trial (IIT) framework, which permits faster, less‑stringent early‑phase studies compared with standard drug review processes in the United States and China.

The lack of transparency and the severe outcomes raise concerns about oversight and safety in the fast‑track pathway, prompting questions about its reliability for future cell and gene‑therapy development.