uniQure Announces Significant Presence at Upcoming American Society of Gene and Cell Therapy (ASGCT) Virtual Annual Meeting

~ 22 Presentations at ASGCT, Including New Preclinical Data on Gene Therapy Candidates, Highlight uniQure’s Industry-Leading Research and Technology Capabilities ~

LEXINGTON, Mass. and AMSTERDAM, April 28, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 22 data presentations, of which five are oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2020 Annual Meeting being held May 12-15.

"uniQure’s significant scientific presence at this year’s ASGCT Annual Meeting demonstrates the robustness of our research capabilities and our excellence in developing novel technologies and commercial-scale gene therapy manufacturing,” stated Sander van Deventer, executive vice president of research and product development at uniQure. “We are very pleased to present new preclinical data on our gene therapy candidates for hemophilia A, spinocerebellar ataxia type 3, Fabry disease and data on our advances in technology and manufacturing.”

Specific details on uniQure’s virtual oral presentations at ASGCT include:

Title: One-Time Intrathecal Administration of AAV5-miATXN3 in Non-Human Primates
Date and Time: Wednesday May 13, 4:15 p.m. EDT/ 10:15 p.m. CET

Title: A Novel NAGA Variant Designed to be Non-immunogenic In Humans and Provide Broad Cross-Correction in Fabry Disease
Date and Time: Thursday May 14, 4:15 p.m. EDT/ 10:15 p.m. CET

Title: A Single Administration of AAV5-hFIX in Newborn, Juvenile and Adult Mice Leads to Stable hFIX Expression up to 18 Months after Dosing
Date and Time: Thursday May 14, 4:15 p.m. EDT/ 10:15 p.m. CET

Title: Characterizing Next-Generation Baculovirus Transduction Processes - A Quality by Design-based Approach for AAV Manufacturing
Date and Time: Friday May 15, 10:45 a.m. EDT/ 4:45 p.m. CET

Title: Clearance of Vector DNA From Bodily Fluids in Patients with Severe or Moderate-Severe Hemophilia B Following Systemic Administration of AAV5-hFIX and AAV5-hFIX Padua
Date and Time: Friday May 15, 11:15 a.m. EDT/ 5:15 p.m. CET

The following presentations were approved for poster presentation:

• Lipid Nanoparticle Pre-Treatment Improves rAAV Diffusion in the Primate Liver and Enables an Increase of Therapeutic Transgene Expression
   
• Prevalence and Avidity Assessment of Pre-existing Neutralizing Antibodies (NABs) Against Adeno-Associated Virus (AAV) Vector Serotypes 2, 5 And 8 Analyzed in The Serum Of 300 Healthy Donors