We at uniQure are fully committed to the clinical development of AMT-130, the first AAV gene therapy to be investigated in Huntington’s disease. We are deeply appreciative of the support that we’ve received from the community and for the pioneering individuals who have volunteered to be a part of our clinical studies. Only through such collaboration can we strive to develop safe and effective treatments to modify the course of Huntington’s disease.
Huntington’s disease (HD) is a rare, fatal, neurodegenerative genetic condition that affects motor function and leads to behavioral symptoms and cognitive decline in adults, resulting in total physical and mental deterioration over a 12 to 15-year period. Huntington’s disease affects approximately 75,000 people in the U.S., Europe and UK.
Huntington’s disease (HD) is an inherited condition that causes the progressive breakdown of brain cells. Buildup of mutant huntingtin protein is thought to cause the disease. The striatum is a core structure of the brain that is first affected in people with Huntington’s. This structure is critical for motor function and reward- and goal-oriented behavior. Loss of brain cells in the striatum leads to the following problems:
- Motor function issues
- Cognitive dysfunction
- Psychiatric disturbances
Despite the discovery of the gene that causes HD, there are no therapies available to treat the disease, delay onset, or slow progression of a patient’s decline.
AMT-130 is an investigative gene therapy for Huntington’s disease that is intended to silence the huntingtin gene, with the goal of inhibiting the production of the mutant protein.
We are currently conducting Phase I‑II clinical trials of AMT-130 in the U.S. and Europe (Learn more).
In September 2025, we announced positive topline data from the study, analyzing clinical outcomes for 29 treated patients (17 high-dose and 12 low-dose) with up to 36 months of follow-up (12 patients per dose at month 36). The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant (p=0.003) 75 percent slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 36 months compared to an external control. The study also met a key secondary endpoint in achieving statistically significant (p=0.033), 60 percent slowing of disease progression as measured by Total Functional Capacity (TFC) at 36 months compared to an external control.
We believe these data support that AMT-130 has the potential to meaningfully slow disease progression – offering long-awaited hope to individuals and families impacted by this devastating disease.
In November 2025, we announced that we received feedback from the U.S. Food and Drug Administration (FDA) during a pre-Biologics License Application (BLA) meeting regarding AMT-130.
Read more about AMT-130 and why we believe it has the potential to alter the course of this disease.
You can read more about uniQure’s gene therapy approach to HD in this brochure.
