Uniqure disclosed Phase 1/2 data from its AMT‑130 gene‑therapy program for Huntington disease showing a sustained reduction in disease progression. The intracerebral AAV‑delivered microRNA therapy produced an estimated 75% slowing of clinical decline at three years versus historical controls in treated patients, per company reports and a Nature/industry summary. AMT‑130 uses RNA interference to lower mutant huntingtin expression and the trial tracked clinical scales and biomarker signals. The result, if validated in randomized studies, would mark a rare long‑term disease‑modifying signal in Huntington’s and accelerate regulatory and investment interest in neurodegenerative gene therapies.