IP Library › Granted Patent US 12,357,663
Granted Patent B2
US 12,357,663 · App. 18/149,151 · Granted Jul 15, 2025

Intrathecal delivery of recombinant adeno-associated virus encoding methyl-CpG binding protein 2

Inventors: Brian K. Kaspar (Westerville, OH); Kevin Foust (Columbus, OH)
Assignees: NATIONWIDE CHILDREN'S HOSPITAL, INC.; OHIO STATE INNOVATION FOUNDATION
A61K35/76A61K9/0019A61K9/0085A61P25/00C07K14/4702C12N7/00C12N2750/14132C12N2750/14143
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Quick Facts
Patent No.
US 12,357,663
App. No.
18/149,151
Granted
Jul 15, 2025
Kind
B2
Abstract

Methods and materials for intrathecal delivery of recombinant Adeno-associated virus 9 (rAAV9) encoding Methyl-CpG binding protein 2 (MECP2) are provided. Use of the methods and materials is contemplated, for example, for the treatment of Rett syndrome.

Claims (24)

1. A recombinant adeno-associated virus (rAAV) vector comprising a gene cassette comprising a promoter consisting of the nucleotide sequence of SEQ ID NO: 2, an SV40 intron, a polynucleotide sequence encoding a methyl-CpG-binding protein 2 (MECP2) and a synthetic polyadenylation signal, wherein the gene cassette is flanked by a mutant AAV2 inverted terminal repeat and a wild type inverted terminal repeat.

2. The rAAV vector of claim 1 , wherein the viral vector is self-complementary (scAAV).

3. The rAAV vector of claim 1 , wherein the rAAV is rAAV9.

4. The rAAV vector of claim 1 , wherein the polynucleotide sequence encoding the MECP2 comprises SEQ ID NO: 3.

5. A recombinant adeno-associated virus (rAAV) vector comprising a gene cassette comprising nucleotides 151-2558 of SEQ ID NO: 1.

6. A virus comprising the rAAV9 vector of claim 3 .

7. The virus of claim 6 , further comprising one or more capsid proteins.

8. A pharmaceutical composition comprising the rAAV vector of claim 1 .

9. A method of treating Rett syndrome in a patient comprising intrathecally administering the rAAV vector of claim 1 to a patient in need thereof.

10. The method of claim 9 further comprising intrathecally administering iohexol, iobitridol, iomeprol, iopamidol, iopentol, iopromide, ioversol or ioxilan, or mixtures of two or more thereof.

11. A virus comprising the rAAV vector of claim 4 .

12. The virus of claim 11 , further comprising one or more capsid proteins.

13. A pharmaceutical composition comprising the rAAV vector of claim 4 .

14. A method of treating Rett syndrome in a patient comprising intrathecally administering the rAAV vector of claim 4 to a patient in need thereof.

15. The method of claim 14 further comprising intrathecally administering iohexol, iobitridol, iomeprol, iopamidol, iopentol, iopromide, ioversol or ioxilan, or mixtures of two or more thereof.

16. A virus comprising the rAAV vector of claim 5 .

17. The virus of claim 16 , further comprising one or more capsid proteins.

18. A pharmaceutical composition comprising the rAAV vector of claim 5 .

19. A method of treating Rett syndrome in a patient comprising intrathecally administering the rAAV vector of claim 5 to a patient in need thereof.

20. The method of claim 19 further comprising intrathecally administering iohexol, iobitridol, iomeprol, iopamidol, iopentol, iopromide, ioversol or ioxilan, or mixtures of two or more thereof.

21. The rAAV vector of claim 5 , wherein the viral vector is self-complementary (scAAV).

22. The rAAV vector of claim 5 , wherein the rAAV is rAAV9.

23. A virus comprising the rAAV9 vector of claim 22 .

24. The virus of claim 23 , further comprising one or more capsid proteins.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 13, 2025
From: KASPAR, BRIAN K.
To: NATIONWIDE CHILDREN'S HOSPITAL INC.
Reel/Frame 070208/0347 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 13, 2025
From: FOUST, KEVIN
To: OHIO STATE INNOVATION FOUNDATION
Reel/Frame 070208/0374 →
Continuity (3)
Continuation 16461837
Provisional Application 62423618 · Nov 17, 2016
Related Publication 20230321164A1 · Oct 12, 2023
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