Breakthrough UK Gene Therapy Slows Huntington's Disease Progression by 75%

Landmark Achievement in Huntington's Treatment

A groundbreaking gene therapy trial conducted in the United Kingdom has achieved a significant milestone, successfully slowing the progression of Huntington's disease by an average of 75% in patients over a three-year period. This marks the first time a treatment has demonstrated such a substantial impact on the devastating neurodegenerative illness. The positive results were announced on September 24, 2025, bringing renewed hope to thousands affected by the condition.

The experimental treatment, known as AMT-130, was developed by the Dutch biotech company uniQure. The trial was led by researchers from University College London's (UCL) Huntington's Disease Centre, with Professor Sarah Tabrizi, the centre's director, and Professor Ed Wild serving as principal investigators.

Understanding the Gene Therapy: AMT-130

The Phase I/II clinical trial involved 29 patients treated across sites in the UK and the US. Those in the high-dose group experienced a 75% slowing of disease progression after 36 months, as measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS), which assesses motor, cognitive, and functional measures. Significant improvements were also noted in other key indicators, including Total Functional Capacity and reduced levels of neurofilaments, a biomarker for brain cell damage.

The gene therapy works by inactivating the mutant protein, called huntingtin, which is responsible for causing Huntington's disease. A harmless virus is used as a vector to deliver a specially designed strand of DNA into neurons. This DNA then instructs the brain cells to block the production of the toxic huntingtin protein.

Surgical Delivery and Future Prospects

The administration of AMT-130 involves a complex surgical procedure where the therapy is delivered directly into the brain. This entails drilling a small hole in the patient's head and inserting a microcatheter to infuse the treatment over approximately 12 to 20 hours. Despite the invasive nature, researchers reported that patients tolerated the procedure well with manageable side effects.

Professor Tabrizi expressed immense optimism, stating, 'We now have a treatment for one of the world's more terrible diseases. This is absolutely huge. I'm really overjoyed.' The breakthrough offers the potential for patients to preserve daily function, remain in employment longer, and significantly slow disease progression. There is also hope that administering the therapy at an earlier stage could prevent symptoms from occurring.

uniQure plans to submit an application for accelerated approval to the US Food and Drug Administration (FDA) early next year, with subsequent applications expected in the UK and Europe. While these early results are promising, experts emphasize the need for larger, late-stage trials to further confirm the long-term benefits and safety of AMT-130. Approximately 8,000 people in the UK are currently living with Huntington's disease.