Promising Gene Therapy Hints at Slowing Huntington's Disease Progression

A small trial of uniQure’s AMT-130 gene therapy in 29 Huntington’s disease patients showed reduced production of the mutant huntingtin protein, slower cognitive decline, and lower levels of neurofilament light in cerebrospinal fluid over three years, suggesting a potential disease-modifying effect. Results were reported in a press release and have not yet undergone peer review, but they point to a possible treatment that could be used earlier in the disease course, while researchers continue to identify early biomarkers and expand the treatment window before motor symptoms appear.