Following the pivotal Phase I/II study results showcasing a 75% slowdown in Huntington’s disease progression, UniQure is preparing to seek FDA approval for its gene therapy AMT-130 in early 2026. The treatment targets the mutated huntingtin gene and is the first potential disease-modifying therapy for this neurodegenerative condition. Positive safety and efficacy data from three years of follow-up underpin this regulatory milestone, opening new hope for affected patients worldwide.