Towards AAV5-mediated Gene Therapy for Hemophilia A with a Factor IX Variant that functions independently of FVIII Ying Poi Liu1, Vanessa Zancanella1, Betty Au1, Paula Montenegro-Miranda1, Martin de Haan1, Viola J.F. Strijbis2, Mettine H.A. Bos2, Karin Huber3, Joachim Schwäble3, Erhard Seifried3, Pavlina Konstantinova1, Sander Van Deventer1 1uniQure Biopharma B.V., Amsterdam, The Netherlands; 2Div. of Thrombosis and Hemostasis, Einthoven Laboratory for Vascular and Regenerative Medicine, Leiden University M ...

uniQure Sustained mutant huntingtin lowering in Huntington disease minipigs mediated by AAV5-miHTT (AMT-130) gene therapy Astrid Vallès, PhD Senior Scientist, Biomarker Lead - New Therapeutic Target Discovery ASGCT 22nd Annual Meeting - May 1, 2019 Forward-looking Statements This presentation contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "exp ...

uniQure Delivering Gene Therapy to Patients SVB Leerink March 1, 2019 D E L I V E R I N G G E N E T H E R A P Y T O P A T I E N T S M A R C H 2 0 1 9 | 2 Forward-looking Statements This presentation contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to," "may," "plan," "potential," "predict," "project," "s ...