IP Library › Granted Patent US 10,385,338
Granted Patent B2
US 10,385,338 · App. 15/549,072 · Granted Aug 20, 2019

Treatment of facioscapulohumeral dystrophy

Inventors: Julie Dumonceaux (Joinville-le-pont, FR); Thomas Voit (London, GB); Virginie Mariot (Paris, FR)
Assignees: ASSOCIATION INSTITUT DE MYOLOGIE; INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE; SORBONNE UNIVERSITE; CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
C12N15/113A61K31/713C12N2310/13C12N2310/3183C12N2310/346C12N2310/532
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Quick Facts
Patent No.
US 10,385,338
App. No.
15/549,072
Granted
Aug 20, 2019
Kind
B2
Abstract

The present invention relates to nucleic acids, compositions and methods for the treatment of facioscapulohumeral dystrophy.

Claims (20)

1. A viral vector comprising a DUX4 decoy nucleic acid, wherein said DUX4 decoy nucleic acid comprises one or more DUX4 binding sites.

2. The viral vector according to claim 1 , wherein said DUX4 decoy nucleic acid comprises 2, 3, 4, 5 or more than 5 DUX4 binding sites.

3. The viral vector according to claim 1 , wherein the DUX4 binding site(s) is(are) of the sequence TAAYYBAATCA (SEQ ID NO:1) or TAAYBYAATCA (SEQ ID NO:2).

4. The viral vector according to claim 2 , wherein the DUX4 binding site(s) is(are) of the sequence TAAYYBAATCA (SEQ ID NO:1) or TAAYBYAATCA (SEQ ID NO:2).

5. The viral vector according to claim 1 , wherein the DUX4 binding site(s) is selected in the group consisting of TAACCCAATCA (SEQ ID NO:3), TAATTTAATCA (SEQ ID NO:4), TAATCCAATCA (SEQ ID NO:5) and TAATTGAATCA (SEQ ID NO:6).

6. The viral vector according to claim 2 , wherein the DUX4 binding site(s) is selected in the group consisting of TAACCCAATCA (SEQ ID NO:3), TAATTTAATCA (SEQ ID NO:4), TAATCCAATCA (SEQ ID NO:5) and TAATTGAATCA (SEQ ID NO:6).

7. The viral vector according to claim 1 , wherein the decoy nucleic acid comprises a sequence of any one of SEQ ID NO:7 to 28.

8. The viral vector according to claim 1 , wherein the decoy nucleic acid consists of a sequence of any one of SEQ ID NO:7 to 28.

9. The vector according to claim 1 , wherein the viral vector is a lentiviral vector or an AAV vector.

10. The vector according to claim 2 , wherein the viral vector is a lentiviral vector or an AAV vector.

11. The vector according to claim 3 , wherein the viral vector is a lentiviral vector or an AAV vector.

12. The vector according to claim 4 , wherein the viral vector is a lentiviral vector or an AAV vector.

13. The vector according to claim 5 , wherein the viral vector is a lentiviral vector or an AAV vector.

14. The vector according to claim 6 , wherein the viral vector is a lentiviral vector or an AAV vector.

15. The vector according to claim 7 , wherein the viral vector is a lentiviral vector or an AAV vector.

16. The vector according to claim 8 , wherein the viral vector is a lentiviral vector or an AAV vector.

17. A pharmaceutical composition comprising the viral vector of claim 1 and a pharmaceutically or physiologically acceptable carrier.

18. A method of treating FSHD in a subject in need thereof, comprising the administration of a therapeutically effective amount of the viral vector according to claim 1 to said subject.

19. A method of treating FSHD in a subject in need thereof, comprising the administration of a therapeutically effective amount of the viral vector according to claim 7 to said subject.

20. A method of treating FSHD in a subject in need thereof, comprising the administration of a therapeutically effective amount of the viral vector according to claim 8 to said subject.

Assignments (2)
MERGER AND CHANGE OF NAME Recorded Jun 19, 2019
From: UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6); UNIVERSITE PARIS-SORBONNE (PARIS IV)
To: SORBONNE UNIVERSITE
Reel/Frame 049514/0536 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 10, 2019
From: DUMONCEAUX, JULIE; MARIOT, VIRGINIE; VOIT, THOMAS
To: ASSOCIATION INSTITUT DE MYOLOGIE OF FRANCE; CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE; INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE; UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6)
Reel/Frame 049146/0415 →
Priority Claims (1)
EP 15305184 · Feb 6, 2015 · regional
Continuity (1)
Related Publication 20180016577A1 · Jan 18, 2018