NextFin news, Scientists from University College London (UCL) and collaborators in the Netherlands and the United States reported on Wednesday, September 24, 2025, that an experimental gene therapy called AMT-130 has successfully slowed the progression of Huntington's disease by 75% in clinical trials.
The trial involved 29 patients with Huntington's disease, a devastating inherited neurodegenerative disorder that causes progressive loss of motor control, cognition, and psychiatric symptoms. The gene therapy was administered via a complex 12- to 18-hour brain surgery, where a modified virus delivers therapeutic DNA directly into the brain. This DNA produces microRNA that disables the production of the mutant huntingtin protein responsible for brain cell death in affected individuals.
Results from the 36-month follow-up of 12 patients who received a high dose of AMT-130 showed a statistically significant 75% reduction in disease progression compared to historical data. Specifically, patients on the therapy experienced a mean decline on the Unified Huntington's Disease Rating Scale of 0.38, versus 1.52 in untreated patients. Additionally, total functional capacity, which measures daily living independence, declined 60% slower in treated patients.
Professor Sarah Tabrizi, director of the UCL Huntington's Disease Centre and lead investigator, said, "We never in our wildest dreams would have expected a 75% slowing of clinical progression." She emphasized the potential for this therapy to transform patient outcomes and is already working on prevention trials for gene carriers without symptoms.
Professor Ed Wild, also from UCL, highlighted that some trial participants remained stable over time, with one medically retired patient able to return to work. The therapy appears to save brain cells, as indicated by reduced neurofilament levels in spinal fluid, a biomarker of neuronal death.
Jack May-Davis, a 30-year-old barrister's clerk carrying the faulty gene, expressed hope, stating, "This absolutely incredible breakthrough has left me overwhelmed. It does allow me to think my life could be that much longer." Vicky Delicata, whose family is affected, also welcomed the news, hoping for NHS availability soon.
The gene therapy was developed by the biotech company uniQure, which plans to apply for regulatory approval in the United States in early 2026, with submissions in Europe and the UK to follow. UniQure's Chief Medical Officer, Dr. Walid Abi-Saab, said the company is eager to discuss the data with regulators and aims to bring the treatment to market next year.
Huntington's disease affects approximately 75,000 people across the UK, US, and Europe, with many more carrying the gene mutation that will eventually cause the disease. Currently, no disease-modifying treatments exist, making this breakthrough a significant milestone in neurodegenerative disease therapy.
The trial's success marks the first time a treatment has demonstrably slowed Huntington's disease progression, offering hope for improved quality of life and extended functional years for patients worldwide.
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