Uniqure reported three‑year follow‑up from its AMT‑130 program in Huntington’s disease showing a 75% slowing of progression on the cUHDRS (composite Unified Huntington's Disease Rating Scale) versus a historical control group. AMT‑130 is an AAV‑delivered gene therapy encoding an artificial microRNA designed to lower huntingtin expression. The Phase 1/2 data involve direct intracranial administration and underscore durable disease‑modifying signals in early patients. Developers say the approach uses a compact mir‑451‑based design to avoid variable Dicer processing and off‑target effects. The results will drive regulatory discussions on pivotal study design and the broader role of one‑time gene therapies for neurodegenerative diseases. Clarification: AMT‑130 uses RNA interference delivered by AAV to reduce mutant huntingtin protein; the mir‑451 format is a non‑canonical microRNA strategy intended to improve safety and predictability.