Gene Therapy Shows Promise in Slowing Huntington’s Disease Progression
BOSTON, MA – A novel gene therapy, AMT-130, has demonstrated meaningful potential in slowing the progression of Huntington’s disease, a currently incurable and devastating neurological disorder, according to recently released Phase III trial data. the treatment, developed by UniQure, resulted in a 75% reduction in disease worsening in measures of lower body function compared to those who did not receive the therapy. Furthermore, the study showed a 60% slowdown in the rate of overall functional decline – a key study objective.
Huntington’s disease, a hereditary condition, causes the progressive breakdown of nerve cells in the brain, leading to motor, cognitive, and psychiatric disturbances. Affecting an estimated 30,000 Americans and a similar number in Europe, the disease typically manifests in adulthood and gradually robs individuals of their ability to walk, talk, and think. Researchers are hopeful that AMT-130 could represent the first approved treatment to modify the course of this relentless illness, offering patients the possibility of maintaining independence and quality of life for longer. UniQure plans to submit an accelerated approval request to the U.S. Food and Drug administration (FDA) in early 2026,with similar applications anticipated in the United Kingdom and Europe.
the trial, led by researchers at University College London (UCL), indicated that a single dose of AMT-130 appeared to be sufficient to achieve these benefits. “For patients,AMT-130 has the potential to preserve their daily functions,to keep them at work longer and to significantly slow down the progression of the disease,” stated Professor Sarah Tabriz of UCL,the principal scientific advisor for the trial.
Professor Ed Wild, principal investigator at the UCL Huntington Disease Test Site, hailed the results as transformative. “These results change everything,” he said. “On the basis of this data, it seems likely that AMT-130 is the first authorized treatment to slow down Huntington’s disease, which really changes the world.”
The therapy was also reported to be “generally tolerated” by participants, with a “manageable safety profile.” While further testing and regulatory approvals are required before widespread availability, the data represents a major step forward in the fight against Huntington’s disease and offers renewed hope to patients and families impacted by this debilitating condition.
