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.
The applications are for the company's investigational gene therapy candidate for adults with hemophilia A.
The data presented at ISTH reflected marked improvements in FVIII half-life amongst study participants.
uniQure's gene therapy candidate AMT-061 is being developed for individuals with severe and moderately severe hemophilia B.
The final data was presented for the first time at a symposium at the XXVII Congress of the International Society on Thrombosis and Haemostasis (ISTH).
Much of the data was gleaned from the HAVEN clinical trial program.
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