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US 2022/0090042 A1Published application
Factor ix polypeptide mutant, its uses and a method for its production
AssigneeuniQure biopharma B.V.
InventorPaolo SIMIONI
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Abstract
Disclosed are a modified FIX (Factor IX) polypeptide comprising a leucine, cysteine, aspartic acid, glutamic acid, histidine, lysine, asparagine, glutamine or tyrosine in position 338; pharmaceutical preparations containing said modified FIX polypeptide; a nucleotide sequence coding for the modified FIX polypeptide; and a method for producing the modified FIX polypeptide.
Claims3
A gene therapy method for the prophylaxis and therapy of hemophilia B, the method comprising:
- administering to a human individual in need thereof an adeno-associated virus (AAV) viral vector, wherein the AAV viral vector comprises a nucleotide sequence encoding a modified Factor IX (FIX) polypeptide,
- wherein the modified FIX polypeptide comprises the amino acid sequence of SEQ ID NO:14, and
- wherein the amino acid at position 148 of SEQ ID NO:14 is either an alanine or a threonine.
Dependencies are inferred from the claim text.