uniQure announced compelling 36-month data from its Phase I/II trial of AMT-130 gene therapy for Huntington’s disease, demonstrating a 75% slowing of disease progression on the composite Unified Huntington’s Disease Rating Scale. This statistically significant effect was greater than anticipated and encompasses multiple functional domains, including motor and cognitive measures. The study involved U.S. and European cohorts, underscoring the therapy's broad applicability. uniQure plans to file a Biologics License Application to the FDA in early 2026, aiming for launch later that year. University College London experts hailed the data as transformative for patients and families, spotlighting a critical step toward a licensed disease-modifying treatment for Huntington’s disease.