Etranacogene dezaparvovec is UniQure's investigational gene therapy candidate for patients with severe and moderately severe hemophilia B.
NHF and HFA have submitted another letter to Bayer requesting information on this serious event.
UniQure plans to exceed its original recruitment goal by "over-enrolling" an additional six trial participants by the end of September.
Marstacimab would represent an alternative approach to standard factor VII or factor IX replacement therapy.
Orphan Drug Designation is awarded by the U.S. FDA to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States.
The new blood-based screening tool represents an alternative to a standard clot-based FVIII test which cannot accurately determine inhibitor levels in patients receiving Hemlibra®.
Much of the data for this analysis was drawn from the HAVEN clinical trial program, which was designed to assess the efficacy and safety of emicizumab in hemophilia A patients with and without FVIII inhibitors.
The company is developing gene therapies for both hemophilia A and B, and for several other chronic conditions.
Concizumab is being investigated for the prevention of bleeding episodes in hemophilia A patients, and in hemophilia A/B patients with inhibitors.
Both AAV vectors and genome editing novel technologies are being brought to bear in this unique and experimental gene therapy.
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