Exclusive: A couple paid more than $800,000 for a gene-editing therapy for their daughter. She died, and it wasn’t made public

Key Points:

• A clinical trial at Shanghai’s Xinhua Hospital involved a 6-year-old girl with a rare genetic mutation receiving an experimental gene-editing therapy, funded partly by over $860,000 from her family.
• Seven days after the therapy infusion, the girl died from a severe immune reaction linked to the treatment, a fatality that was never publicly reported or disclosed in the related Nature publication.
• The trial proceeded under a regulatory loophole without national approval, raising concerns about lax oversight, inadequate risk communication to the family, and ignoring safety signals from preclinical studies.
• Experts reviewing the case have called for a full investigation of the trial data and funding transparency, suggesting that the Nature paper may need to be retracted due to ethical and scientific issues.
• The girl's family has publicly shared their story to demand accountability from the researchers and institutions involved, expressing dismay over the concealed risks and lack of safeguards.