uniQure's Huntington's gene therapy effect narrows at four years

uniQure released four-year data on AMT-130 (ifezuntirgene inilparvovec) on 29 September 2026. It is a one-time gene therapy injected into the brain to lower huntingtin. The company filed for accelerated US approval earlier in September, on 36-month data. The new cut shows the effect holding on one measure and shrinking on the other.

At 36 months, the 15 high-dose patients showed 80% slower progression on the composite cUHDRS scale than a propensity-matched external control (p=0.005). At 48 months, with 12 high-dose patients, that figure was 44% (p=0.144), which is not statistically significant. Total Functional Capacity, the primary measure of the confirmatory study, held up better: 61% slower decline at 48 months (p=0.008). Neurofilament light in spinal fluid, a marker of nerve damage, was 6% below baseline at 36 months and 4% above it at 48.

Five high-dose participants (17%) had serious inflammation-related adverse events in the central nervous system, and all of them resolved.

The comparison is with an external control, not a randomised arm, in groups of about a dozen patients. According to BioPharma Dive, uniQure’s shares fell about 40% on the day because of the durability question.