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

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uniQure N.V. (NASDAQ: QURE) announced five data presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting from May 11-14. The presentations will focus on gene therapy advancements targeting hemophilia and Huntington’s disease, highlighting the AAV5 vector's efficacy amid pre-existing neutralizing antibodies. Notable sessions include two oral presentations on the Phase 3 HOPE-B trial and miQURE technology's proof-of-concept for liver targeting. These sessions aim to showcase uniQure's innovation in gene therapy for severe medical conditions.

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LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 28, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (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 pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

uniQure Contacts:

FOR INVESTORS:		FOR MEDIA:
Maria E. Cantor	Chiara Russo	Tom Malone
Direct: 339-970-7536	Direct: 617-306-9137	Direct: 339-970-7558
Mobile: 617-680-9452	Mobile: 617-306-9137	Mobile:339-223-8541
m.cantor@uniQure.com	c.russo@uniQure.com	t.malone@uniQure.com

FAQ

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What is the focus of uniQure's presentations at ASGCT 2021?

uniQure's presentations will focus on gene therapy advancements for hemophilia and Huntington’s disease, emphasizing the effectiveness of the AAV5 vector.

When will uniQure present at the ASGCT Annual Meeting?

uniQure will present from May 11-14, 2021, with specific sessions scheduled on May 11, 12, and 13.

What are the key topics of the oral presentations by uniQure?

The oral presentations will cover the Phase 3 HOPE-B trial for Etranacogene Dezaparvovec and a proof-of-concept for miQURE technology in liver disease.

What new technology does uniQure plan to discuss?

uniQure plans to discuss the miQURE technology, which shows potential for effective gene targeting in liver-based diseases.

Which disease areas is uniQure targeting with its gene therapies?

uniQure is targeting hemophilia B, Huntington's disease, Fabry disease, and spinocerebellar ataxia Type 3, among others.