Contact Us

Learn more about the HOPE-B trial on ClinicalTrials.gov HOPE-B Trial
Send us an email with your questions uniQureHOPE-B@uniqure.com


  • 113 Hartwell Avenue
    Lexington, MA 02421

Get involved

Join us in the development of a new therapy for hemophilia B

I am a healthcare provider I am a patient
For more information, please provide the following:
REFERENCES
  1. 1. Genetics Home Reference - health: Do all gene mutations affect health and development? https://ghr.nlm.nih.gov/primer/mutationsanddisorders/neutralmutations. Accessed January 15, 2019.
  2. 2. National Human Genome Research Institute. Learning about hemophilia. https://www.genome.gov/20019697/learning-about-hemophilia/. Accessed January 15, 2019.
  3. 3. Genetics Home Reference - treatment: How are genetic conditions treated or managed? https://ghr.nlm.nih.gov/primer/consult/treatment. Accessed January 15, 2019.
  4. 4. Rogers GL, Herzog RW. Gene therapy for hemophilia. Front Biosci. 2015;20:556-603.
  5. 5. Miesbach W, Meijer K, Coppens M, et al. Gene therapy with adeno-associated virus vector 5-human Factor IX in adults with hemophilia B. Blood. 2018;131(9):1022-1031
  6. 6. Leebeek F, MeijerK, Coppens M, et al. Reduction in annualized bleeding and factor IX consumption up to 2.5 years in adults with severe or moderate-severe hemophilia B treated with AMT-060 (AAV5-hFIX) gene therapy. Blood. 2018;132:3476.
  7. 7. uniQure announces initial topline data from dose-confirmation study of AMT-061 in patients with hemophilia B [news release]. Lexington, MA: uniQure; November 15, 2018. https://tools.eurolandir.com/tools/pressreleases/GetPressRelease/?ID=3536930&lang=en-GB&companycode=nl-qure&v=. Accessed January 15, 2019.
  8. 8. Von Drygalski A, Giermasz A, Castaman G, et al. Phase 2b trial of AMT-061 (AAV5- Padua hFIX): Translation into humans of an enhanced gene transfer vector for adults with severe or moderate-severe hemophilia B. Haemophilia. 2019:25(Suppl. 1);25–34.

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The company is developing several gene therapies for the treatment of patients with liver/metabolic, central nervous system, and other severe genetic diseases.

This website is maintained by uniQure Inc., having its offices at 113 Hartwell Avenue, Lexington, MA 02421 USA.