Uniqure has announced positive top-line data from the pivotal Phase I/II study of its gene therapy AMT-130 for Huntington's disease, demonstrating a statistically significant 75% slowing in disease progression at 36 months compared to an external control. This first-of-its-kind one-time treatment delivers microRNA designed to silence the mutated huntingtin gene, addressing the neurodegenerative condition that currently has no approved therapies. With plans to submit for FDA approval in early 2026, this represents a potential breakthrough for patients suffering from this debilitating disorder. The trial measured efficacy using composite Unified Huntington's Disease Rating Scale (cUHDRS) and Total Functional Capacity (TFC).