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Patents/USRE050288

USRE050288No. USRE050288reissueGranted 2/4/2025

Claims (21)

Claim 1 (Independent)

1. A pharmaceutical composition comprising: a modified recombinant FIX (factor IX) polypeptide comprising at least 70% identity to SEQ ID NO: 2 and a leucine in position 338 of SEQ ID NO: 2; the modified recombinant FIX polypeptide in an amount suitable to provide a daily dosage comprised between 0.1 μg/kg and 400 μg/kg body weight, and at least one pharmaceutically acceptable excipient.

Claim 6 (Independent)

6. A sequence of nucleotides encoding a modified FIX (Factor IX) polypeptide comprising at least 70% identity to SEQ ID NO: 2 and a leucine at position 338 of SEQ ID NO: 2.

Claim 8 (Independent)

8. A nucleotide sequence encoding a modified FIX (Factor IV) polypeptide comprising 100% identity to SEQ ID NO: 2 except for an alanine at position 148 and a leucine at position 338 of SEQ ID NO: 2 in combination with positions corresponding to positions 34098, 34099 and 34100 of a sequence having accession number K02402 (GenBank) having a triplet selected from the group consisting of: TTA, UUA, TTG, UUG, CTT, CUU, CTC, CUC, CTA, CUA, CTG, and CUG , GAT, GAU and GAC .

Show 18 dependent claims
Claim 2 (depends on 1)

2. The pharmaceutical composition according to claim 1 , wherein the polypetide comprises at least 90% identity to SEQ ID NO: 2.

Claim 3 (depends on 1)

3. The pharmaceutical composition according to claim 1 formulated for oral, parenteral or rectal administration or suited to administration by inhalation or insufflation.

Claim 4 (depends on 1)

4. A method of treatment, the method comprising administering to an individual in need thereof the pharmaceutical composition according to claim 1 .

Claim 5 (depends on 4)

5. The method according to claim 4 wherein the administering is performed to treat at least one coagulopathy in the individual.

Claim 7 (depends on 6)

7. The sequence of nucleotides according to claim 6 , wherein the nucleotide sequence has at least 90% homology with the sequence having accession number K02402 (GenBank).

Claim 9 (depends on 6)

9. A nucleic acid comprising the nucleotide sequence according to claim 6 .

Claim 10 (depends on 9)

10. The nucleic acid according to claim 9 , the nucleic acid comprising a promoter in operational linkage with said nucleotide sequence.

Claim 11 (depends on 9)

11. A vector comprising the nucleic acid according to claim 9 .

Claim 12 (depends on 6)

12. A pharmaceutical composition comprising the sequence of nucleotide according to claim 6 .

Claim 13 (depends on 9)

13. A method for producing a modified FIX (Factor IX) polypeptide, the method comprising expressing the modified FIX polypeptide by means of a nucleic acid according to claim 9 .

Claim 14 (depends on 6)

14. A method to perform gene therapy, the method comprising administering to an individual in need thereof the nucleotide sequence according to claim 6 via a vector configured for gene therapy.

Claim 15 (depends on 14)

15. The method according to claim 14 , wherein the administering is performed to treat at least one coagulopathy in the individual.

Claim 16 (depends on 8)

16. The nucleotide sequence of claim 8 , wherein the triplet is selected from the group consisting of CTC and CTG.

Claim 17 (depends on 8)

17. The nucleotide sequence of claim 8 , wherein the triplet is selected from the group consisting of CUC and CUG.

Claim 18 (depends on 8)

18. The nucleotide sequence of claim 8 , wherein the triplet is CTC.

Claim 19 (depends on 8)

19. The nucleotide sequence of claim 8 , wherein the triplet is CTG.

Claim 20 (depends on 8)

20. The nucleotide sequence of claim 8 , wherein the triplet is CUC.

Claim 21 (depends on 8)

21. The nucleotide sequence of claim 8 , wherein the triplet is CUG.

Full Description

No description text available for this patent.

Citations

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