UniQure NV announced promising Phase I/II clinical trial results for its gene therapy AMT-130 targeting Huntington’s disease, demonstrating a significant 75% slowing in disease progression after 36 months. The trial met primary and key secondary endpoints using the composite Unified Huntington’s Disease Rating Scale and Total Functional Capacity. This one-time therapy, delivered via delicate brain surgery, represents a potential breakthrough for a currently incurable neurodegenerative disorder, offering substantial hope for patients and families. Experts from University College London highlighted the study as the most convincing evidence to date of meaningful disease modification in Huntington’s disease.