UniQure Therapeutics reported positive topline results from its pivotal Phase I/II trial of AMT-130 for Huntington’s disease, meeting its primary endpoint with a statistically significant 75% slowing of disease progression at 36 months compared to matched controls. Secondary endpoints, including functional capacity, also showed benefit. This gene therapy approach uses microRNA to silence mutant huntingtin protein, conferring disease-modifying potential. These data validate the therapy as a promising first-in-class treatment for a currently unmet need in neurodegeneration.