uniQure's gene therapy for hemophilia B

UniQure, Pfizer updates hint at gene therapy potential in hemophilia B

  • The first patient has been treated in a 50-subject Phase 3 clinical trial, HOPE-B, evaluating uniQure's (NASDAQ:QURE) gene therapy AMT-061 in patients with severe and moderately severe hemophilia B.
  • The primary endpoint is Factor IX activity 26 weeks after dosing.
  • The estimated primary completion date is March 2020.
  • AMT-061 has Breakthrough Therapy status in the U.S. and PRIME status in Europe.

Read More ... https://www.biopharmadive.com/news/uniqure-pfizer-hemophilia-b-gene-therapy-updates/568809/


We are proud to announce the first online guided tour of a Hemophilia Treatment Center (HTC).

HTC Tour