The Day the Definition of "Incurable" Changes - The Impact of 75% Progression Suppression: AMT-130 Gene Therapy Transforming Huntington's Disease

uniQure, a Netherlands/US-based company, has achieved the primary endpoint at the 36-month mark in a Phase 1/2 trial of its Huntington's disease gene therapy, AMT-130, targeting early-stage patients. In the high-dose group, the composite Unified Huntington's Disease Rating Scale (cUHDRS) showed a 75% reduction in disease progression compared to external controls, and the Total Functional Capacity (TFC) measure indicated a 60% reduction. The safety profile was generally acceptable, with adverse events related to the surgical procedure of MRI-guided intracranial administration reported as reversible. This news has generated strong anticipation among researchers and the patient community, with significant reactions from media commentators and investors. However, there are still many unresolved issues, such as the small number of subjects, limitations of external controls, long-term safety and durability, scope of application, and costs. uniQure is considering a US application in 2026, with future peer-reviewed publications, conference presentations, and discussions with regulatory authorities being key factors.