uniQure in the NEWS On September 24, 2025, uniQure N.V. (QURE) - a gene therapy company advancing transformative therapies for patients with severe medical needs, announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease. The study met its prespecified primary . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

The United States FDA granted Breakthrough Therapy Designation to AMT-061. The European Medicines Agency gave it access to the Priority Medicine (PRIME) regulatory initiative. Gene therapy firm, uniQure (QURE), announced updated clinical data in patients treated in the ongoing Phase 2b study of AMT-061; an investigational AAV5-based gene therapy containing a patent-protected  FIX9-Padua (Factor 9) for the treatment of patients with severe and moderately severe hemophilia B. The announced data, which …