Overview

• In the high-dose cohort, clinical progression slowed by roughly 75% over 36 months versus matched natural-history controls, while the low dose showed limited effect.
• Cerebrospinal-fluid neurofilament light, a marker of neuronal damage, fell by an average of 8.2% at three years after an initial post-surgical spike.
• The Phase 1/2 study treated 29 participants via a single 12–18 hour bilateral striatal neurosurgery using a viral vector to deliver microRNA targeting huntingtin.
• Most adverse events were associated with the surgical procedure and were reported to resolve, with the therapy not halting disease progression entirely.
• Findings are preliminary and unpeer-reviewed from a small study using external comparators, and investigators are expanding to larger multicenter trials as UniQure pursues accelerated approval in 2026.