IP Library › Patent Application 14981981
Patent Application
App. No. 14/981,981

FACTOR IX POLYPEPTIDE MUTANT, ITS USES AND METHOD FOR ITS PRODUCTION

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Quick Facts
Patent No.
US None
App. No.
14/981,981
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 (12)

1 . A modified recombinant FIX (factor IX) polypeptide comprising at least 70% identity to SEQ ID NO: 2 or SEQ ID NO: 1 and an aspartic acid at a position corresponding to position 338 of SEQ ID NO: 2.

2 . A polypeptide according to claim 1 , and having an identity of at least 90% SEQ ID NO: 2 or SEQ ID NO: 1.

3 . A method to perform replacement therapy, the method comprising administering to an individual in need thereof the modified FIX polypeptide according to claim 1 .

4 . The method according to claim 3 for the treatment of at least one coagulopathy.

5 . The method according to claim 3 wherein the modified FIX polypeptide is administered in a daily dosage comprise between 0.1 μg/kg and 400 μg/kg.

6 . A pharmaceutical composition comprising a modified recombinant FIX polypeptide according to claim 1 , and at least one pharmaceutically acceptable excipient.

7 . The pharmaceutical composition according to claim 6 comprising the modified FIX polypeptide in an amount suitable to provide a daily dosage comprised between 0.1 μg/kg and 400 μg/kg.

8 . The pharmaceutical composition according to claim 6 formulated for oral, parenteral or rectal administration or suited to administration by inhalation or insufflation.

9 . A method for producing a modified FIX polypeptide according to claim 1 , whereby the modified FIX polypeptide is expressed by means of a nucleic acid comprising a nucleotide sequence encoding a modified FIX polypeptide according to claim 1 .

10 . The method according to claim 9 wherein the nucleotide sequence has at least 90% identity with the sequence having accession number K02402 (GenBank).

11 . The method according to claim 10 wherein the nucleotide sequence comprises in positions corresponding to positions 34098, 34099 and 34100 a triplet selected in a group consisting of: GAT, GAU, GAC.

12 . The method according to claim 9 wherein the nucleic acid comprises a promoter in operational linkage with said nucleotide sequence.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 20, 2017
From: SIMIONI, PAOLO
To: UNIQURE BIOPHARMA B.V.
Reel/Frame 043909/0855 →