Huntington's Gene Therapy Still Appears to Slow Daily Function Decline at Four Years, but Main Score Misses Significance

Four years after a single brain infusion, people with Huntington's disease who received a high dose of uniQure's experimental gene therapy AMT-130 were still losing day-to-day abilities more slowly than matched patients who never received it, the company reported September 29. But the trial's main measure, a combined score of thinking, movement, and function, no longer showed a statistically significant difference. The mixed result comes as the FDA decides whether to formally accept the company's application.

Update Note: This article reports new four-year trial results for AMT-130 (ifezuntirgene inilparvovec) following MedicalDaily's earlier coverage of uniQure's FDA filing. The newest figures come from uniQure's announcement of the four-year data, released September 29, 2026. The data run through a June 30, 2026, cutoff.