Gene Therapy Slows Huntington's Disease Progression by 75% in Landmark Trial

A groundbreaking gene therapy treatment has successfully slowed the progression of Huntington's disease by 75% in a recent clinical trial, offering unprecedented hope for patients with the fatal inherited brain disorder. The therapy, developed by uniQure and involving researchers from institutions like University College London, marks the first time a treatment has significantly altered the disease's course. This breakthrough could potentially extend patients' good quality of life by decades and may even prevent symptoms if administered early.

Huntington's disease is a devastating neurodegenerative condition that damages nerve cells, leading to features akin to dementia, Parkinson's, and motor neurone disease. Until now, treatments have focused solely on managing symptoms, with no options to halt or reverse the relentless decline. The successful trial represents a monumental shift in the fight against this illness, which typically manifests in adulthood and is invariably fatal.

The experimental therapy, known as AMT-130, utilizes cutting-edge genetic medicine combining gene therapy and gene silencing technologies. It is delivered directly into the brain through a delicate surgical procedure, where it works by reducing the production of the toxic huntingtin protein responsible for the disease. This one-time treatment aims to permanently lower the harmful protein levels in the brain.

Results from the Phase I/II trial, involving 29 patients, indicated a 75% slowing of clinical progression over three years for those receiving a high dose of the treatment. This means that a decline typically expected in one year could now take four years, according to researchers. Professor Sarah Tabrizi, director of the University College London Huntington's Disease Centre and lead researcher, described the findings as "spectacular," adding, "We never in our wildest dreams would have expected a 75% slowing of clinical progression." Some patients in the trial have reportedly returned to work or maintained mobility when they were expected to lose it.

uniQure plans to meet with the U.S. Food and Drug Administration (FDA) later this year and aims to file a Biologics License Application in early 2026, with a potential launch in the U.S. later that year. While the treatment involves complex surgery and is anticipated to be expensive, its potential to fundamentally transform the lives of approximately 75,000 individuals affected by Huntington's disease in the U.S., Europe, and UK offers a new horizon of possibilities.