Uniqure and UCL scientists announce positive results for new treatment
Patients treated with the investigational gene therapy AMT-130 experienced a 75% reduction in disease progression over 36 months, compared with matched participants from the long-running Enroll-HD natural history study, according to new data released by uniQure.
This is the first drug trial to report sustained, statistically significant slowing of Huntington’s disease progression. uniQure plans to submit an application for accelerated approval to the US FDA in early 2026, with UK and European submissions to follow.
The Phase I/II trial enrolled 29 participants, 12 of whom received a high dose of AMT-130 and now have 36 months of follow-up. Disease progression was assessed using the composite Unified Huntington’s Disease Rating Scale (cUHDRS), Total Functional Capacity, and additional motor and cognitive endpoints. All demonstrated clinically meaningful benefit versus the external comparator cohort.
Biomarker data reinforced these findings: treated patients showed a reduction in cerebrospinal fluid neurofilament light (NfL) levels, contrasting with the 20–30% rise typically observed over three years in untreated Huntington’s disease. Investigators say this points to a genuine disease-modifying effect.
Safety data indicate AMT-130 is generally well tolerated, with a manageable profile following neurosurgical delivery.
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Professor Sarah Tabrizi (UCL Huntington’s Disease Research Centre and UK Dementia Research Institute, UCL) described the results as “the most convincing evidence in the field to date,” highlighting AMT-130’s potential to preserve function and extend working life. Professor Ed Wild (UCL Huntington’s Disease Centre) called the findings “world-changing,” adding that patients’ stability over time is “not something we usually see in Huntington’s disease.”
If approved, AMT-130 would be the first licensed therapy to slow Huntington’s disease progression, more than three decades after discovery of the causative gene.
Additional details are available at Gene therapy appears to slow Huntington’s disease progression | UCL News – UCL – University College London and www.clinicaltrials.gov (NCT0543017, NCT04120493)
