UniQure’s investigational gene therapy, AMT-130, achieved a 75% reduction in disease progression for Huntington’s disease after three years, representing the most compelling data in the field so far. This pivotal trial success propels the therapy closer to becoming the first FDA-approved treatment modifying the course of Huntington’s disease. Market reaction was strong, with shares surging following the announcement. The therapy exemplifies the potential of single-treatment gene therapies in rare neurodegenerative diseases.