UniQure gene therapy slows Huntington’s less at four years than at three

Marcus Bell · 2026-09-29T13:20:08.641Z

The weakening effect at four years, now not even significant, raises real doubts about lasting benefits for patients.

UniQure gene therapy slows Huntington’s less at four years than at three — © IJR

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uniQure on September 29, 2026 announced 48-month and updated 36-month Phase I/II data for AMT-130 (ifezuntirgene inilparvovec) in Huntington's disease. In 12 high-dose patients at 48 months, cUHDRS progression was 44% slower than matched ENROLL-HD external controls, with p=0.144, not statistically significant. Total Functional Capacity showed 61% slowing (nominal p=0.008). Shares dropped 55% premarket. Updated 36-month data for 15 high-dose patients showed 80% cUHDRS slowing (nominal p=0.005) and 67% TFC slowing (nominal p=0.011). Twenty-nine patients were treated (17 high dose, 12 low dose); data cutoff was June 30, 2026. Five high-dose patients had treatment-related CNS inflammation serious adverse events that all resolved. uniQure said matched controls had 53% missing data at 48 months and that control patients who discontinued were progressing faster than those remaining, which the company said may have reduced the estimated treatment effect. Reuters reported the therapy fell short of statistical significance on the main goal. The new results were not included in uniQure's Biologics License Application, which relied on 36-month data from 12 high-dose patients. No approved disease-modifying treatment exists for Huntington's.

UniQure gene therapy slows Huntington’s less at four years than at three

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