On September 29, 2026, uniQure N.V. released updated clinical data for its gene therapy candidate ifezuntirgene inilparvovec (AMT-130) in the treatment of Huntington’s disease. The update covers Phase I/II results at 36 and 48 months with a data cutoff of June 30, 2026. At 36 months, high-dose patients showed an 80% slowing of disease progression based on cUHDRS (p=0.005) and 67% based on TFC (p=0.011). At 48 months, the primary endpoint cUHDRS showed a non-significant 44% slowing (p=0.144), while TFC demonstrated a significant 61% slowing (p=0.008). Post-hoc analysis using prior controls suggested stronger effects at 48 months. The therapy was generally well-tolerated, with one treatment-related serious adverse event of CNS inflammation reported since September 2025. uniQure N.V. is a gene therapy company developing treatments for rare diseases including hemophilia and Huntington’s disease.
