IP Library › Patent Application 16952514
Patent Application
App. No. 16/952,514

EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY

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Quick Facts
Patent No.
US None
App. No.
16/952,514
Abstract

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.

Claims (13)

1 - 26 . (canceled)

27 . An antisense oligomer conjugate of Formula (III):

or a pharmaceutically acceptable salt thereof.

28 . A pharmaceutically acceptable salt of the antisense oligomer conjugate of claim 27 .

29 . The antisense oligomer conjugate of claim 27 , wherein the antisense oligomer is of Formula (IIIA):

30 . The antisense oligomer conjugate of claim 27 , wherein the antisense oligomer is of Formula (IV):

or a pharmaceutically acceptable salt thereof.

31 . A pharmaceutically acceptable salt of the antisense oligomer conjugate of claim 30 .

32 . The antisense oligomer conjugate of claim 30 , wherein the antisense oligomer is of Formula (IVA):

33 . A pharmaceutical composition comprising the antisense oligomer conjugate of claim 27 , or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.

34 . A pharmaceutical composition comprising the antisense oligomer conjugate of claim 29 and a pharmaceutically acceptable carrier.

35 . A pharmaceutical composition comprising the antisense oligomer conjugate of claim 30 and a pharmaceutically acceptable carrier.

36 . A pharmaceutical composition comprising the antisense oligomer conjugate of claim 32 and a pharmaceutically acceptable carrier.