uniQure Presents Preclinical Data Demonstrating Advances in the Distribution of Gene Therapy - Seite 2

Increase of Therapeutic Transgene Expression in the Liver

Valerie 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.com

uniQure Forward-Looking Statements

This 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.