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Breakthrough Gene Therapy Slows Huntington's Disease by 75% : ScienceAlert
Researchers in the United Kingdom say they have successfully trialed what could become the world's first gene therapy for Huntington's disease – a fatal neurodegenerative disorder that is typically inherited.
While the results of the clinical trial are not yet formally published or peer reviewed, principal investigator and neuroscientist Ed Wild from University College London says the gene therapy, called AMT-130, "changes everything." "Trial results come through in numbers and graphs, but behind each data point is an incredible patient who volunteered to undergo major neurosurgery to be treated with the first gene therapy we've ever tested in Huntington's disease," says Wild. The chief medical officer, Walid Abi-Saab, says uniQure is "eager to discuss the data with the Food and Drug Administration (FDA)… later this year, with the goal of submitting a Biologics License Application in the first quarter of 2026."
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