UniQure’s AMT-130 gene therapy delivered remarkable results, showing a statistically significant 75% slowing in Huntington's disease progression over 36 months in high-dose patients, according to recent clinical data. Additional endpoints indicated improvements across motor and cognitive measures. The data have driven UniQure’s stock to a five-year peak and sparked optimism for a first disease-modifying HD therapy. The company plans to submit an FDA Biologics License Application in early 2026 with a potential market launch later that year.