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

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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, 2018

AAV5 Gene Delivery to the Central Nervous System

Raygene 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 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.