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

-- 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 (GLOBE NEWSWIRE) -- 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.