— Preclinical Data Presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Chicago —LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 18, 2018 — uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, presented preclinical data in three poster presentations at this week’s American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Chicago, Illinois. The posters highlight uniQure’s expertise in efficient transduction of target cells within affected organs, showing that uniQure’s modular platform is now broadly applicable to treat patients with both liver and central nervous system (CNS) disorders.
“We are rapidly progressing our understanding and capabilities related to AAV delivery, which have the potential to significantly expand the application of our gene therapies to patients with serious metabolic liver diseases and neurocognitive disorders,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer of uniQure. “We believe our expertise enables us to implement novel technologies that lead to effective transduction of a wide range of target cell types in the CNS and liver. We also demonstrate the ability to express therapeutic proteins throughout the primate liver, which is mandatory for the efficacy of gene therapy for many metabolic diseases. This is an important step toward uniQure’s goal of delivering transformational medicine to patients suffering from genetic diseases.” Selected Highlights from Poster Presentations at ASGCT, May 17-18, 2018AAV5 Gene Delivery to the Central Nervous SystemRaygene Martier, a Ph.D. student in uniQure’s New Therapeutic Targets Discovery group, presented new data demonstrating transduction by AAV5 vectors in different cell types in the CNS, including astrocytes, motor neurons and medium spiny neurons. The data showed that AAV5 achieved a diffuse transduction profile via direct injection in the ventricles of the brain, suggesting that diseases with involvement of deep brain structures can be targeted with AAV5. Based on these data, AAV5 gene therapy may be applicable to a wide range of neurodegenerative diseases where broad coverage of the CNS is desired. Increase of Therapeutic Transgene Expression in the LiverValerie Ferreira, Ph.D., uniQure’s head of immunology, presented recent enhancements to the systemic administration of AAV5 gene therapies that substantially improve transduction in the liver.First, Dr. Ferreira described a method that exactly determines the percentage of hepatocytes transduced following gene therapy in primates and simultaneously quantifies the expression of therapeutic protein for each transduced cell. Using this method, in a separate poster, Dr. Ferreira subsequently showed that pre-treatment with a lipid emulsion prior to AAV5 administration significantly improves the efficacy of AAV5 vector delivery to the liver and enables broader hepatic cell targeting throughout the tissue. By blocking the liver sinusoidal endocytotic capacity, pretreatment with a lipid emulsion significantly increased the percentage of transduced hepatocytes and caused a uniform distribution of the vector throughout the liver tissue. This approach, which can be readily implemented in future clinical trials, represents an important tool for future liver-targeted gene therapies, such as metabolic disorders that require broad transduction of the liver.
Oral Session on Saturday, May 19, 2018
On Saturday, May 19, at 11:45 a.m. CDT, uniQure scientist Anna Majowicz, Ph.D., will deliver an oral presentation examining the relationship between the presence of pre-existing anti-AAV5 neutralizing antibodies and clinical outcomes of AMT-060 in patients with hemophilia B. 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 and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and cardiovascular diseases. www.uniQure.comuniQure 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, our upcoming anticipated milestones, the development of our gene therapy product candidates, the transition to our AMT-061 product candidate, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates. 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, 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 uniQure’s Quarterly Report on Form 10-Q filed on April 30, 2018. 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.uniQure Contacts:FOR INVESTORS:Maria E. Cantor
[email protected] Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
[email protected] FOR MEDIA:Tom Malone
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