UniQure has reported highly promising 36-month clinical data for AMT-130, its gene therapy candidate for Huntington’s disease (HD). The Phase I/II study demonstrated a statistically significant 75% slowing in disease progression on the composite Unified Huntington’s Disease Rating Scale among high-dose patients. Additional efficacy measures showed slowing from 59% to over 100% in various motor and cognitive tests. Dr. Sarah Tabrizi from University College London hailed these results as ‘game-changing,’ reflecting a potential shift toward licensed disease-modifying HD therapies. UniQure plans to submit a Biologics License Application to the FDA in early 2026, targeting a launch later that year.