Overview

• In the Phase 1/2 study of 29 patients in the UK and US, the high-dose AMT-130 group showed about 75% slower clinical decline at 36 months compared with matched natural-history controls.
• Cerebrospinal fluid neurofilament light levels fell on average after treatment, countering the rises typically seen as neurons degenerate.
• The one-time therapy is delivered by a 12–20 hour MRI-guided infusion into the striatum using a viral vector to lower mutant huntingtin production.
• UniQure reports a manageable safety profile with adverse events largely related to the surgical procedure and resolving.
• Results remain preliminary pending peer review and larger controlled trials, with a formal presentation due in October and an FDA accelerated-approval submission planned for early 2026.