A report in STAT detailed the sudden death of a child two and a half days after receiving an experimental brain‑directed gene therapy, triggering concern across academic and commercial gene‑therapy programs. The fatality has prompted investigators and companies to re‑examine safety signals associated with viral vectors engineered to penetrate the central nervous system. Researchers and industry groups say the case has raised questions about vector tropism, dosing, and immune responses in vulnerable pediatric populations. Sponsors developing brain‑targeting adeno‑associated or related viral platforms are re‑reviewing preclinical safety packages and clinical protocols while regulators and IRBs await further data from the case.
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