Deaths and setbacks highlight gene therapy safety debate

Science

Gizmodo

Gene therapy shows life-changing promise but recent adverse cases have renewed safety debates. Science reported a six-year-old girl's death after an experimental gene-editing trial in China, and pharmaceutic firm HuidaGene disclosed a second likely gene-therapy-related death in a boy with Duchenne muscular dystrophy; both used China’s investigator-initiated trial (IIT) pathway. In the United States, doctors at CHOP reported a child developed a brain tumor probably driven by the AAV viral vector used four years earlier. Experts urge more transparency, risk-based development, independent oversight, and faster external review to balance safety with urgent patient needs.

Two deaths in China and a U.S. tumor have raised safety concerns