Gene Therapy Shows Promise for Huntington’s Disease
University College London researchers and developer uniQure reported results from a three‑year, global clinical trial of AMT‑130 — a one‑time, brain‑injected gene therapy — showing that high‑dose recipients (n in trial) had about 75% less disease progression versus standard care on the Unified Huntington’s Disease Rating Scale. The 29‑patient study also demonstrated favorable biomarker changes (neurofilament light protein) and a manageable safety profile, a development that could prompt additional trials and regulatory review for a disease that previously had no disease‑modifying treatments.
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📌 Key Facts
- Trial: three‑year clinical trial conducted at UCL and global sites with 29 Huntington’s patients enrolled
- Efficacy: patients receiving a high dose of AMT‑130 experienced roughly 75% less progression on the Unified Huntington’s Disease Rating Scale compared with standard of care
- Biomarkers & safety: neurofilament light protein (NfL) levels were lower than expected over the trial period and the therapy was described as well‑tolerated with a manageable safety profile