UniQure announced that its experimental gene therapy AMT-130 significantly slowed Huntington’s disease progression by 75% over three years in a Phase 1/2 trial. This pioneering one-time genetic treatment targets the rare neurodegenerative condition characterized by loss of motor and cognitive function. The positive trial outcome supports UniQure’s plan to submit for FDA approval early next year, marking a potential first genetic therapy approval for Huntington’s disease. The data show prolonged motor function preservation compared to matched controls, signaling a promising therapeutic milestone for patients with limited treatment options.