If approved, this would be the first genetic therapy for Huntington\u2019s disease<\/p>\n<\/h3>\n
By I. Edwards HealthDay Reporter<\/b>
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FRIDAY, Sept. 26, 2025 (HealthDay News) \u2014 A new gene therapy has shown promise in slowing the progression of Huntington\u2019s disease, according to early trial results released Wednesday.<\/p>\n
In a Phase 1\/2 study, patients given a high dose of UniQure\u2019s<\/a> experimental therapy AMT-130 experienced a 75% slowing of disease progression after three years, the company said. The therapy also reduced levels of a key marker of brain damage \u2014 neurofilament light protein in spinal fluid \u2014 by an average of 8.2%.<\/p>\n “These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington\u2019s disease,” Dr. Walid Abi-Saab<\/a>, chief medical officer at UniQure, told CNN<\/em>.<\/p>\n The trial, run in partnership with University College London, included 29 patients treated with either a high or low dose of AMT-130 and followed for 36 months.<\/p>\n The treatment was delivered surgically by injecting the therapy directly into the brain\u2019s striatum, an area most affected by Huntington\u2019s.<\/p>\n Most side effects were linked to the surgical procedure and resolved, the company reported.<\/p>\n The findings have not yet been peer-reviewed or published in a medical journal and should be regarded as preliminary.<\/p>\n Huntington\u2019s disease is a rare genetic condition most common among people of European ancestry.<\/p>\n It damages nerve cells in the brain, leading to movement problems, cognitive decline and changes in mood and behavior. There is currently no cure, but treatments can manage some symptoms.<\/p>\n Amy Gray<\/a> of the Huntington\u2019s Disease Society of America (HDSA) remained cautious but hopeful after reviewing the study findings.<\/p>\n \u201cFor the approximate 42,000 Americans and their families living with Huntington\u2019s disease, this is a truly transformative development,\u201d Gray, who was not involved in the research, said in an email to CNN.<\/em><\/p>\n \u201cFor decades, there have been no therapies to slow disease progression \u2014 only treatments to manage symptoms,\u201d she added. \u201cWhile there is still a path to approval through the FDA, this data brings us closer than ever to a future where we can change the course of Huntington\u2019s disease.\u201d<\/p>\n UniQure said it plans to submit its findings to the U.S. Food and Drug Administration (FDA) in early 2026. If approved, AMT-130 could become the first treatment for Huntington\u2019s.<\/p>\n Outside experts said more research is needed, noting the trial was small and relied partly on existing patient data rather than a full placebo-controlled group.<\/p>\n Still, David Rubinsztein<\/a> of the Cambridge Institute for Medical Research called the results \u201cvery promising\u201d and \u201cexciting,” CNN <\/em>reported.<\/p>\n More information<\/strong><\/p>\n The Mayo Clinic has more on Huntington’s<\/a>.<\/p>\n