UniQure reported pivotal Phase 1/2 trial results showing its gene therapy AMT-130 significantly slowed Huntington’s disease progression by 75% over three years. This rare neurodegenerative disorder, marked by relentless brain cell loss, currently lacks effective treatments. The therapy is administered via precise brain surgery and targets the faulty huntingtin gene responsible for disease symptoms. Investigators described the findings as groundbreaking, offering hope for substantially improved patient quality of life. UniQure plans to submit for FDA approval in early 2026, potentially introducing the first genetic therapy for Huntington's.