uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today presented new preclinical data on AMT-130, its proprietary gene therapy candidate for the treatment of Huntington’s disease (HD), at the European Society of Gene and Cell Therapy (ESGCT) 25th Anniversary Congress in Berlin, Germany.
Data from the study demonstrate that following administration of AMT-130 in Huntington’s disease mouse models, significant improvements in both motor-coordination and survival were observed, as well as a dosedependent, sustained reduction in huntingtin protein. AMT-130 comprises an AAV5 vector carrying a DNA cassette encoding an engineered micro RNA (miHTT) that silences the human huntingtin protein. The study on functional improvement and sustained huntingtin lowering was performed by members of uniQure’s research department in collaboration with Charles River Discovery Research Services, Finland.