Four years is a meaningful stretch of follow-up for a one-time experimental gene therapy. It is also long enough for the way a study measures progress to become critically important.
On 29 September 2026, uniQure reported updated results for AMT-130, also called ifezuntirgene inilparvovec, in Huntington’s disease. The treatment uses a surgically delivered viral vector carrying instructions for a small RNA designed to reduce production of the huntingtin protein. This is gene silencing, not an edit that removes the inherited mutation. Company results and treatment description
Two measures, different answers
Among 12 people who had received the higher dose and reached 48 months, the company calculated a 44% slower decline on a composite measure combining movement, thinking and daily function compared with matched people in an external natural-history dataset. That comparison did not meet the conventional threshold for statistical significance (p=0.144). A narrower measure of total functional capacity showed a 61% slower decline, with a nominal p-value of 0.008. Four-year analysis
At three years, an expanded group of 15 higher-dose patients showed a larger difference on the composite score. The company has already submitted a US Biologics License Application based on an earlier three-year analysis of 12 patients. The newly announced figures were not part of that submission. Filing an application does not mean the therapy is approved. Regulatory and cohort details
Why the comparison needs care
These are ongoing Phase I/II studies, and the principal long-term comparison uses matched records of people outside the treatment trial. Matching can reduce obvious differences, but it cannot recreate random assignment. The company says 53% of the matched external control data were missing at four years, which could distort the comparison. A later analysis using an earlier control dataset improved the composite result, but it was performed after the fact and should be read with that limitation. Methods and sensitivity analysis
UniQure also reported treatment-related serious central nervous system inflammation in five higher-dose participants across its programme, which it says resolved. The long follow-up offers an encouraging signal, especially for daily function, alongside real statistical and safety questions. A regulator and a confirmatory study will have to judge whether the effect is reliable. Safety and study details
Featured image: representative image by Milad Fakurian / Unsplash. It does not depict the specific study, facility or equipment described.
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