Uniqure announced a landmark success with its gene therapy AMT-130 demonstrating a 75% slowing of Huntington’s disease progression at 36 months, based on a pivotal Phase I/II study. The therapy targets the mutated huntingtin gene and represents the first gene therapy showing such significant clinical benefit in this rare neurodegenerative disease. Data presented at multiple venues and published analyses affirm AMT-130's potential as a groundbreaking treatment, with plans underway for FDA submission in early 2026. This advance offers long-awaited hope for patients facing this debilitating condition and underscores the transformative impact of genetic medicines in neurology.