IP Library › Granted Patent US 12,377,150
Granted Patent B2
US 12,377,150 · App. 17/804,280 · Granted Aug 5, 2025

Exon skipping oligomer conjugates for muscular dystrophy

Inventors: Marco A. Passini (Cambridge, MA); Gunnar J. Hanson (Cambridge, MA)
Assignee: Sarepta Therapeutics, Inc.
A61K47/549A61K47/645A61P21/00C12N15/113C12N2310/11C12N2310/3233C12N2310/3513C12N2320/35
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Quick Facts
Patent No.
US 12,377,150
App. No.
17/804,280
Granted
Aug 5, 2025
Kind
B2
Abstract

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 45 skipping are described.

Claims (24)

1. A method for treating Duchenne muscular dystrophy (DMD) in a human subject in need thereof, wherein the human subject has a mutation of the dystrophin gene that is amenable to exon 45 skipping, the method comprising administering to the human subject an antisense oligomer conjugate of Formula (IV):

or a pharmaceutically acceptable salt thereof.

2. The method of claim 1 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt.

3. The method of claim 2 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt of Formula (IVA):

4. A method of restoring an mRNA reading frame to induce dystrophin production in a human subject having a mutation of the dystrophin gene that is amenable to exon 45 skipping, the method comprising administering to the human subject an antisense oligomer conjugate of Formula (IV):

or a pharmaceutically acceptable salt thereof.

5. The method of claim 4 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt.

6. The method of claim 5 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt of Formula (IVA):

7. A method for treating Duchenne muscular dystrophy (DMD) in a human subject in need thereof wherein the human subject has a mutation of the dystrophin gene that is amenable to exon 45 skipping, the method comprising administering to the human subject a pharmaceutical composition comprising an antisense oligomer conjugate of Formula (IV):

or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.

8. The method of claim 7 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt.

9. The method of claim 8 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt of Formula (IVA):

10. A method of restoring an mRNA reading frame to induce dystrophin production in a human subject having a mutation of the dystrophin gene that is amenable to exon 45 skipping, the method comprising administering to the human subject a pharmaceutical composition comprising an antisense oligomer conjugate of Formula (IV):

or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.

11. The method of claim 10 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt.

12. The method of claim 11 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt of Formula (IVA):

13. A method of excluding exon 45 from dystrophin pre-mRNA during mRNA processing in a human subject having a mutation of the dystrophin gene that is amenable to exon 45 skipping, the method comprising administering to the human subject a pharmaceutical composition comprising an antisense oligomer conjugate of Formula (IV):

or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.

14. The method of claim 13 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt.

15. The method of claim 14 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt of Formula (IVA):

16. A method of binding exon 45 of dystrophin pre-mRNA in a human subject having a mutation of the dystrophin gene that is amenable to exon 45 skipping, the method comprising administering to the human subject a pharmaceutical composition comprising an antisense oligomer conjugate of Formula (IV):

or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.

17. The method of claim 16 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt.

18. The method of claim 17 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt of Formula (IVA):

Assignments (2)
SECURITY INTEREST Recorded May 7, 2025
From: SAREPTA THERAPEUTICS, INC.
To: JPMORGAN CHASE BANK, N.A. AS ADMINISTRATIVE AGENT
Reel/Frame 071218/0445 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 10, 2024
From: PASSINI, MARCO A.; HANSON, GUNNAR J.
To: SAREPTA THERAPEUTICS, INC.
Reel/Frame 068861/0823 →
Continuity (6)
Continuation 16469104
Provisional Application 62562119 · Sep 22, 2017
Provisional Application 62479177 · Mar 30, 2017
Provisional Application 62443481 · Jan 6, 2017
Provisional Application 62436199 · Dec 19, 2016
Related Publication 20220387601A1 · Dec 8, 2022
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