Advances in Medicine Driven by Innovation
Competitor’s AAV based gene therapies for hemophilia A have failed to demonstrate the lifetime durability and none of the hemophilia A or B gene therapies can treat patients with inhibitors (up to 30% of patients). Gene therapies in development to treat hemophilia A express clotting Factor VIII, which is not natively expressed in hepatocytes targeted by gene therapy. This non-native expression can lead to accumulation of misfolded protein, cell death and loss of expression.
Native expression of FVa gives us durability vs. FVIII, making us the first durable, universal gene therapy for all types of hemophilia (hemophilia A and B) and the first AAV based gene therapy that can treat patients with inhibitors.