Modified plasma clotting factor VIII and method of use thereof
AssigneeAavnergene Inc.
InventorsQizhao Wang, Daozhan Yu
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Abstract
Modified human factor VIII polypeptides with enhanced factor VIII activity are described. In some embodiments, the modified human factor VIII polypeptides comprise one or more amino acid substitutions at positions A20, T21, F57, L69, I80, L178, R199, H212, I215, R269, I310, L318, S332, R378, I610 and/or I661. Such polypeptides and viral vectors encoding such polypeptides may be used for treatment of FVIII deficiencies, such as hemophilia A.
Claims17
A modified human factor VIII (mhFVIII) protein, comprising an A20K amino acid substitution, wherein the position of the substitution refers to the amino acid residue position in SEQ ID NO:3.
A modified human factor VIII (mhFVIII) protein, comprising A20K and T21V amino acid substitutions, wherein the position of the substitution refers to the amino acid residue positions in SEQ ID NO:3.
Dependencies are inferred from the claim text.