UniQure's Huntington's gene therapy four-year trial results expected imminently
Stat News outlines what researchers and clinicians should expect from the forthcoming phase 1/2 readout of UniQure's AMT-130 gene therapy for Huntington's disease.
Stat News reports that UniQure is expected to release four-year data from its closely watched phase 1/2 clinical trial of AMT-130, an AAV-delivered gene therapy for Huntington's disease, before the end of September 2026. The report provides context on what observers regard as meaningful signals at this stage of follow-up.
AMT-130 uses an AAV5 vector to deliver a microRNA designed to reduce expression of the huntingtin (HTT) protein. Earlier readouts at one and two years showed reductions in cerebrospinal fluid neurofilament light chain (NfL), a marker of neuronal damage, and some signals in clinical rating scales, though the trial was not powered for definitive efficacy conclusions at those timepoints.
The four-year data are considered particularly significant because Huntington's disease progression is slow and earlier timepoints offer limited resolution on whether functional decline has been altered. Analysts and patient advocacy groups have highlighted this readout as one of the most consequential near-term milestones in the rare neurological disease pipeline. UniQure previously submitted AMT-130 to the FDA for regulatory review, a development reported in Genetic Current on 3 September 2026.
No results have been published. This item covers anticipated reporting only.
Sources
Read the original reporting — these are the public sources this summary draws from.
-
Primary source Stat News · 2026-09-10STAT+: What to expect from UniQure's upcoming Huntington's gene therapy readout