uniQure’s AAV Gene Therapy AMT-130 Shows Promise for Huntington’s Disease

LEXINGTON, Mass. and AMSTERDAM – September 24, 2025– In a major breakthrough for neurodegenerative diseases, uniQure N.V. has announced positive topline data from its pivotal Phase I/II study of AMT-130, an investigational AAV gene therapy for Huntington’s disease. The company reported that the high-dose AAV treatment significantly slowed disease progression, offering a potential disease-modifying solution for the devastating, inherited disorder.

The study, which used a propensity score-matched external control, met its prespecified primary and key secondary endpoints. At the 36-month mark, patients receiving the high-dose AAV gene therapy demonstrated a statistically significant slowing of progression as measured by both the composite Unified Huntington’s Disease Rating Scale (cUHDRS) and Total Functional Capacity (TFC).

“These groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington’s disease, where an urgent need persists,” said Dr. Sarah Tabrizi, a leading expert in the field. “These data indicate that AMT-130 has the potential to meaningfully slow disease progression—offering long-awaited hope to individuals and families impacted by this devastating disease.”

AAV Therapy Demonstrates Strong Clinical Benefit

The data, with a cutoff of June 30, 2025, showed robust results for the AAV gene therapy:

• cUHDRS: A statistically significant 75% slowing of disease progression (p=0.003) was observed.
• TFC: The therapy achieved a statistically significant 60% slowing of disease progression (p=0.033).
• Supportive Biomarker: A mean reduction in cerebrospinal fluid neurofilament light protein (CSF NfL), a key biomarker of neurodegeneration, further supported the positive clinical findings.

The company believes the consistent results observed in the high-dose group, compared to the variable trends in the low-dose group, reflect a dose-dependent response to the AAV-mediated treatment.

The AAV gene therapy was delivered in a single, one-time administration using MRI-guided neurosurgery directly into the striatum. According to uniQure’s Chief Medical Officer, Dr. Walid Abi-Saab, these findings provide “important evidence supporting one-time, precision-delivered AAV gene therapies for the treatment of neurological disorders.”

Future Outlook

AMT-130 has been granted both Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations by the FDA, reflecting the high unmet medical need and the promising early clinical data. uniQure plans to meet with the FDA later this year to discuss the data and aims to submit a Biologics License Application (BLA) in the first quarter of 2026. This potential approval could mark a paradigm shift in the treatment of Huntington’s disease, moving from symptom management to a potentially curative, one-time genetic intervention using an AAV delivery platform.