Lexington, MA and Amsterdam, the Netherlands, November 4, 2020 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs,today announced that four data presentations, including one oral presentation and three poster presentations, will be delivered at the 62nd American Society of Hematology (ASH) Annual Meeting, which will be held December 5-8 as a virtual event.
“We expect to have a strong presence at the virtual ASH annual meeting next month, and look forward to having long-term follow-up data presented from our hemophilia B gene therapy studies, including two years of follow-up on the Phase IIb clinical trial of etranacogene dezaparvovec (AMT-061) and up to five years of follow-up from the Phase I/II clinical trial of AMT-060,” stated Matt Kapusta, chief executive officer at uniQure. “We also remain on track to announce top-line data from the pivotal HOPE-B study of etranacogene dezaparvovec before the end of this year, either at ASH or through a company presentation.”
Oral Presentation
Title: Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial
Presenter: Annette von Drygalski, M.D., PharmD
Session Name: 801. Gene Editing, Therapy and Transfer I
Date: Monday, December 7, 2020
Presentation Time: 2:45 p.m. ET (11:45 a.m. PT)
Poster Presentations
Title: AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years
Session Name: 801. Gene Editing, Therapy and Transfer: Poster III
Date: Monday, December 7, 2020
Presentation Time: 10:00 a.m. - 6:30 p.m. ET (7:00 a.m. - 3:30 p.m. PT)
Title: A Single Administration of AAV5-hFIX in Newborn, Juvenile and Adult Mice Leads to Stable hFIX Expression up to 18 Months after Dosing
Session Name: 801. Gene Editing, Therapy and Transfer: Poster II
Date: Sunday, December 6, 2020
Presentation Time: 10:00 a.m. - 6:30 p.m. ET (7:00 a.m. - 3:30 p.m. PT)
Title: Examining the Hemophilia Disability Paradox
Session Name: 904. Outcomes Research—Non-Malignant Conditions: Poster II
Date: Sunday, December 6, 2020
Presentation Time: 10:00 a.m. - 6:30 p.m. ET (7:00 a.m. - 3:30 p.m. PT)
The conference abstracts were made available today and can be accessed through this link: ASH abstracts.