uniQure Inc.: uniQure Announces Presentations at the Upcoming 27th Congress of the International Society on Thrombosis and Haemostasis (ISTH)

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, June 21, 2019(NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that five data presentations, of which three are oral presentations, will be featured at the upcoming 27^th congress of the International Society on Thrombosis and Haemostasis (ISTH), taking place in Melbourne, Australia from July 6 - 10, 2019.

"The data to be presented at ISTH showcase our gene therapy expertise in hemophilia B with both long-term safety and durability from our ongoing Phase 1-2 study of AMT-060 , and up to 9 months of safety and FIX activity in the Phase IIb dose-confirmation study of AMT-061," stated Matt Kapusta, chief executive officer at uniQure. "We also are very pleased to have an oral presentation on our novel gene therapy approach to treat hemophilia A and look forward to presenting these data at the conference."

Specific details on uniQure's presentations at ISTH include:

• Title: AMT-061 (AAV5-Padhua hFIX variant) an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Follow-up up to 9 Months in a Phase 2b Trial (OC 01.1)
  Oral Session Title: Gene Therapy Clinical I
  Date and Time: Saturday July 6, 1:00 - 1:15 p.m. AEST/ Friday July 5, 11:00 p.m. ET
  Location: Melbourne Room 2

• Title: Stable Expression of FIX and Maintained Reductions in Bleeding and Factor IX Consumption Following AMT-060 Gene Therapy with up to 3.5 Years of Follow-up in Adults with Severe or Moderate-Severe Hemophilia B (OC 01.4)
  Oral Session Title: Gene Therapy Clinical I
  Date and Time: Saturday July 6, 1:45 - 2:00 p.m. AEST/ Friday July 5, 11:45 p.m. ET
  Location: Melbourne Room 2

• Title: AAV5-mediated Gene Therapy for Hemophilia A with Factor IX Variant that Functions Independently of FVIII (OC 22.3)
  Oral session title: Gene Therapy Basic I
  Date and Time: Sunday July 7, 3:15 - 3:30 p.m. AEST/ Sunday July 7, 01:15 a.m. ET
  Location: Melbourne Room 1

• Title: No Evidence of Germline Transmission of Vector DNA Following Intravenous Administration of AAV5-hFIX to Male Mice (PB 0303)
  Poster Date and Time: Sunday, July 7, 6:30 - 7:30 p.m. AEST/ Sunday July 7, 4:30 a.m. ET
  Location: Exhibition Hall

• Title: Low Predicted Immunogenicity Risk Associated with FIX Variants that can Promote Coagulation in the Absence of FVIII: in vitro and in silico assessments (PB 0301)
  Poster Date and Time: Sunday, July 7, 6:30 - 7:30 p.m. AEST/ Sunday July 7, 4:30 a.m. ET
  Location: Exhibition Hall

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 and partnered gene therapies to treat patients with hemophilia, Huntington's disease and other severe genetic diseases. www.uniQure.com

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