uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI’s 15th Annual Huntington’s Disease Therapeutics Conference – GlobeNewswire

~ Up to Two Years of Follow-up in Large Transgenic Huntingtons Disease Model Demonstrates Stable mHTT Protein Lowering ~

~ Novel Preclinical Data Demonstrates Successful Lowering of Pathogenic Exon 1 Fragment ~

~ Additional Data Demonstrates the Potential of MRS as Imaging Biomarker for Huntingtons Disease Gene-Therapy Studies ~

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Feb. 27, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the presentation of multiple new preclinical data on AMT-130, its investigational AAV gene therapy for the treatment of Huntingtons disease (HD), at the 15th Annual CHDI Huntingtons disease Therapeutics Conference in Palm Springs, California.

Our data presentations at CHDI illustrate the increasing potential of AMT-130 to target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species and show meaningful activity using the presence of extracellular vesicles as a potential biomarker, stated Sander van Deventer, M.D., Ph.D., executive vice president, research & product development of uniQure. In addition, we highlight the use of magnetic resonance spectroscopy as a potentially important imaging biomarker to measure the restoration of target tissue. Collectively, these findings represent a robust package of new preclinical data to better inform how researchers and clinicians pursue a much-needed treatment for this devastating disease.

Four scientific abstracts submitted by uniQure researchers were accepted for presentation at the conference, of which one is an oral presentation to be featured today. Important findings across several preclinical studies presented at the conference include the following:

Translatable Biomarkers in Gene Therapy for Huntington Disease: Learnings from Pre-clinical Studies

Secreted Therapeutics: Monitoring Durability of microRNA-based Gene Therapies in Huntingtons disease

Lowering the Pathogenic Exon 1 HTT Fragment by AAV5-miHTT Gene Therapy

Exploring the Effects of Intrastriatal AAV5-miHTT Lowering Therapy on Neuronal Function, MRS Signal and Mutant Huntingtin Levels in the Q175FDN Mouse Model of Huntingtons disease

The uniQure data presentations featured at CHDI are available on the investor page of the Companys website, http://www.uniQure.com

About Huntingtons DiseaseHuntingtons disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene, that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntingtons disease, there are no therapies to delay the onset or to slow the diseases progression.

About uniQure uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. http://www.uniQure.com

uniQure Forward-Looking StatementsThis press release 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," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether AMT-130 will target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species or show meaningful activity using the presence of extracellular vesicles as a potential biomarker, and whether magnetic resonance spectroscopy will be an important imaging biomarker to measure the restoration of target tissue. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQures Quarterly Report on Form 10-Q filed on October 28, 2019. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

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uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI's 15th Annual Huntington's Disease Therapeutics Conference - GlobeNewswire