Brain‑penetrant gene therapy safety questioned after child’s fatal outcome — field reassesses risk

A fatal adverse event in the first human dosed with a newly engineered brain‑penetrant viral vector has raised safety concerns across gene‑therapy programs aimed at delivering treatments to the central nervous system. STAT reported the child’s death days after dosing and outlined the scientific community’s reassessment of brain‑targeting vector risks, the limits of preclinical models, and implications for ongoing trials and regulatory scrutiny.