Breakthrough: gene therapy slows Huntington's disease by 75%
New gene therapy slows Huntington's disease by 75% and gives patients and families hope for a better future.
Published on September 25, 2025
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For the first time, researchers have successfully developed a gene therapy that can significantly slow the progression of the devastating Huntington's disease. In a clinical trial, the treatment led to a statistically significant 75% delay in disease progression after 3 years, as measured by cognitive and motor tests. This was announced by UniQure, an Amsterdam-based company, in a press release. It offers patients and their families unprecedented hope for a better quality of life. The treatment is expected to be approved in the US in 2026, with a possible European launch thereafter.
AMT-130: A promising gene therapy
The experimental gene therapy, known as AMT-130, developed by UniQure, has shown promising results in slowing the progression of Huntington's disease. The treatment, which is administered via a delicate 12- to 18-hour brain surgery, aims to reduce the levels of the toxic protein that causes the disease. In the crucial Phase I/II study, a high dose of AMT-130 showed a statistically significant 75% delay in disease progression after 36 months, compared to an external control group. This was measured using the composite Unified Huntington's Disease Rating Scale (cUHDRS), an instrument that evaluates patients' cognitive and motor functions. These results represent a significant step forward in the treatment of this debilitating disease.
Improvement in functional capacity and stabilization of biomarkers
In addition to the significant delay in disease progression, the study also showed a significant improvement in patients' Total Functional Capacity (TFC) after 36 months, with a 60% delay. TFC measures a patient's overall functional capacity, including their ability to perform daily activities. In addition, average levels of neurofilaments (NfL) in the cerebrospinal fluid remained below baseline after 36 months. Neurofilaments are structural proteins that are released when brain cells are damaged, and their stabilization indicates that the treatment protects brain cells. These findings suggest that AMT-130 not only slows the symptoms of Huntington's disease but may also reduce the underlying neurodegeneration.
Future plans and potential preventive treatments
The study's results have been received with great enthusiasm by both the medical community and the Huntington's disease community. Professor Sarah Tabrizi, director of the University College London Huntington's Disease Center, described the results as “spectacular” and stated that they had never dared to dream of a 75% delay in clinical progression.
UniQure plans to submit a Biologics License Application (BLA) to the FDA in the United States in the first quarter of 2026, with the aim of bringing the treatment to market later that year. In addition, discussions will be initiated with authorities in the United Kingdom and Europe to make the treatment available there as well.
The impact of Huntington's disease and the promise of AMT-130
Huntington's disease is a devastating genetic disorder that affects brain cells and leads to a combination of dementia, Parkinson's disease, and motor neuron disease. The first symptoms usually appear between the ages of 30 and 40 and often lead to death within two decades. The ability to slow disease progression by 75% and significantly improve quality of life is a huge step forward. In addition, the treatment has been shown to spare brain cells, paving the way for future preventive treatments.
