uniQure Inc.: uniQure Announces Presentations at Upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 28, 2021(NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that five data presentations, of which two are oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting being held May 11-14.

"Our presentations at ASGCT showcase our gene therapy expertise in hemophilia and Huntington's disease, as well as advantages of the AAV5 vector in dosing through pre-existing neutralizing antibodies," stated Ricardo Dolmetsch, Ph.D.president of research and development at uniQure. "In addition, we look forward to presenting preclinical data demonstrating the potential of the miQURE technology used in our CNS product candidates to be effective in liver-based diseases as well."

Specific details on uniQure's presentations at ASGCT include:

• Title: Clinical Outcomes in Patients With and Without Pre-existing Neutralizing Antibodies to the Vector: 6-Month Data from the Phase 3 HOPE-B Gene Therapy Trial of Etranacogene Dezaparvovec (Abstract #88)
  Oral Session Title: Immune Responses to AAV Vectors
  Date and Time: Wednesday, May 12, 6:15 - 6:30 p.m. EDT

• Title: First Proof-of-Concept of miQURE^TM Based Gene Targeting in the Liver: Lipid Lowering and Atherosclerosis Suppression by AAV-miQURE^TM-Mediated ANGPTL3 Targeting (Abstract #156)
  Oral Session Title: Cardiovascular and Pulmonary Gene Therapy
  Date and Time: Thursday, May 13, 6:30 - 6:45 p.m. EDT

• Title: Lowering the Pathogenic Exon 1 HTT Fragment by AAV5-miHTT Gene Therapy (Abstract #446)
  Poster Session Title: Oligonucleotide Therapeutics
  Date and Time: Tuesday, May 11, 8:00 - 10:00 a.m. EDT

• Title: Presence of Pre-Existing Anti-Adeno-Associated Virus (AAV) Serotype 5 Neutralizing Antibodies (NABs) in Serum of Huntington Disease (HD) Patients Was Not Associated with Detectable Anti-AAV5 Nabs in Cerebrospinal Fluid (CSF) (Abstract #736)
  Poster Session Title: Immunological Aspects of Gene Therapy and Vaccines
  Date and Time: Tuesday, May 11, 8:00 - 10:00 a.m. EDT

• Title: Dynamics, Features and Cross-Reactivity of IgG Pool Induced after AAV5 Gene Therapy for Hemophilia B (Abstract #741)
  Poster Session Title: Immunological Aspects of Gene Therapy and Vaccines
  Session Date and Time: Tuesday, May 11, 8:00 - 10:00 a.m. EDT

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 pipelineof proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

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