uniQure has announced that its experimental gene therapy, AMT-130, appeared to slow the progression of Huntington’s disease over three years in patients with early-stage disease.
Though complete data remain unpublished, the company’s announcement underscores the potential and the cautious optimism surrounding the results.
Huntington’s disease, a devastating inherited condition, causes progressive degeneration of nerve cells in the brain, leading to movement, cognitive, and psychiatric decline. For decades, efforts to arrest or reverse its course have failed. Against that background, uniQure’s results stand out.
The company said the trial involved a high-dose cohort of 12 patients who underwent neurosurgical delivery of AMT-130.
AMT-130 is designed to reduce the expression of the mutant huntingtin gene by targeting a specific microRNA sequence. UniQure states that those treated exhibited significantly slower decline across multiple clinical, functional, and cognitive measures when compared with an externally matched control group drawn from global registries. Biomarker evidence also suggested a drop in neurofilament light protein (NfL), consistent with reduced neuronal injury.
uniQure stated that AMT-130 is intended as a one-time intervention, rather than repeated dosing. The company frames these early results as laying the groundwork for Phase 3 trials, with an emphasis on long-term safety, broader patient cohorts, and reinforcing the durability of effect. Though the therapy’s delivery demands complex brain surgery, uniQure says the potential for durable slowing of disease is a compelling trade-off.
Dr Sophie Andrews, clinical neuropsychologist at the University of the Sunshine Coast, said, “To detect such a large effect in such a small patient group is incredibly promising… Although preliminary, these positive findings across multiple measures after three years indicate that this really could be a promising, long-lasting treatment.”
Professor Bryce Vissel, of St Vincent’s Hospital, Sydney and UNSW, said, “This is the first public claim of statistically significant slowing on both the composite clinical and functional scales over three years in a human Huntington’s study… It requires independent scrutiny and, ideally, confirmation.”
Associate Professor Lyndsey Collins-Praino of the University of Adelaide described the 75 per cent slowing in disease progression in the high-dose group as “very exciting,” while cautioning that long-term safety, access, and cost remain important unknowns.
At the same time, uniQure announced a capital raise that bolsters its cash runway and underpins investor confidence in AMT-130’s potential. The company told investors the proceeds will fund upcoming Phase 3 studies, expand manufacturing capabilities, and support other pipeline programs.