The following is an excerpt from a press release from uniQure. Read the full press release here.
uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. AMT-061 has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.
“We are very pleased to have successfully administered AMT-061 to the first patient enrolled in the HOPE-B pivotal trial and mark this as a milestone for the field in advancing a potential one-time treatment for patients with hemophilia B,” said Robert Gut, M.D., Ph.D., chief medical officer of uniQure. “I am extremely proud of the efforts made by the uniQure team to advance this study, and we plan to complete full patient enrollment in this study before the end of this year.”
“Our mission in hemophilia B has always been to be first to market with a best-in-class gene therapy,” said Matt Kapusta, chief executive officer. “With the initiation of the dosing phase of our pivotal study for AMT-061 and the data generated to date in our Phase IIb study, we believe that we are on track toward our goal of developing the first gene therapy that provides durable, functionally-curative benefits to nearly all patients with hemophilia B, without the complications associated with capsid-related immune responses or the need for immunosuppression therapy.”
Read the full press release here.