On 29 September 2026, uniQure announced additional data from its ongoing phase 1/2 studies, comparing updated 36-month and 48-month outcomes with propensity score-matched external controls (Enroll-HD natural history dataset).
Disease progression, assessed using the composite Unified Huntington’s Disease Rating Scale, was slowed by 80% at 36 months (n = 15; nominally statistically significant) and 44% at 48 months (n = 12; not statistically significant). Disease progression was slowed significantly at both 36 and 48 months for Total Functional Capacity, a secondary endpoint. For the high-dose group, mean cerebrospinal fluid neurofilament light chain was 6% below baseline at 36 months (n = 14) and 4% above baseline at 48 months (n = 11).
The 36-month data from 12 participants who received the high dose (announced 24 September 2025) form the basis for the recent regulatory submissions.

For details of all ongoing and completed EHDN-endorsed studies, click here.