US2021238573A1PendingUtilityA1

Factor ix polypeptide mutant, its uses and a method for its production

Assignee: UNIQURE BIOPHARMA B VPriority: Sep 15, 2008Filed: Mar 26, 2021Published: Aug 5, 2021
Est. expirySep 15, 2028(~2.1 yrs left)· nominal 20-yr term from priority
Inventors:Paolo Simioni
A61P 7/04A61K 38/4846C12Y 304/21022C12N 9/644
73
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Claims

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.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . An adeno-associated virus (AAV) vector comprising:
 a nucleic acid encoding a modified FIX polypeptide, the modified FIX polypeptide comprising SEQ ID NO:2 with the exception of a leucine at position 338 of SEQ ID NO:2 and an alanine at position 148 of SEQ ID NO:2.

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