uniQure Presents New Preclinical Data in SCA3, Fabry Disease, and Hemophilia A at the ASGCT Annual Meeting

~ Substantial Presence with 22 Data Presentations Highlighting Breadth of Research, Technology and Manufacturing Platform ~

~ New Non-Human Primate Data Demonstrate Feasibility of Intracisternal
Administration of AMT-150 for SCA3 ~

~ New Data Show a Single Administration of AAV5-hFIX in Newborn Mice
Led to Long-Term, Stable hFIX Expression ~

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 14, 2020 (GLOBE NEWSWIRE) --  uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, presented new preclinical data on its gene therapy candidates AMT-150 for Spinocerebellar Ataxia type 3, AMT-190 for Fabry disease, and AMT-180 for the treatment of Hemophilia A. The data are featured in presentations at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.  uniQure is delivering a total of 22 data presentations at the meeting, which is taking place virtually from May 12 to May 15.

“Our collective presence at ASGCT showcases the breadth of uniQure’s gene therapy expertise and leadership, including our research capabilities and excellence in developing and manufacturing novel gene therapies,” stated Matt Kapusta, chief executive officer at uniQure. “We are happy to share new preclinical data on our gene therapy candidates, as well as our innovations in technology and manufacturing.”

The uniQure presentations in Spinocerebellar Ataxia type 3 (SCA3) show a continuation of strong proof-of-concept data in mice and other preclinical models, as well as encouraging new data in non-human primates (NHPs). Additionally, new data related to AAV biology show that a single administration of AAV5-hFIX in newborn mice led to stable hFIX expression up to 18 months after dosing.

AMT-150 for Spinocerebellar Ataxia type 3 (SCA3)

SCA3, also known as Machado-Joseph disease, is caused by a CAG-repeat expansion in the ATXN3 gene that results in an abnormal form of the protein ataxin-3. People with SCA3 experience brain degeneration that results in movement disorders, rigidity, muscular atrophy and paralysis. There is currently no treatment available that slows the progressive course of this lethal disease. AMT-150 is a one-time, intracisternally-administered, AAV gene therapy incorporating the Company’s proprietary miQURE silencing technology that is designed to halt ataxia in early manifest SCA3 patients.  AMT-150 is currently in pre-investigational new drug enabling studies.