UniQure reported that its intracranial AAV‑delivered microRNA therapy AMT‑130 produced a 75% slowing of Huntington’s disease progression versus historical controls at three years in a Phase 1/2 cohort. The one‑time brain infusion reduced mutant huntingtin protein levels and delivered durable clinical signal in treated patients, according to the company’s published data. The result supports continued development but will require larger, randomized studies to confirm clinical benefit and to evaluate long‑term safety of CNS AAV delivery. Clarification: AMT‑130 employs RNA interference delivered by AAV to lower toxic huntingtin protein expression.