US2021169919A1PendingUtilityA1
Exon skipping oligomer conjugates for muscular dystrophy
Est. expiryDec 19, 2036(~10.4 yrs left)· nominal 20-yr term from priority
A61K 31/712A61P 21/00C12N 15/113A61K 47/645C12N 2310/111A61K 31/7125
70
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Claims
Abstract
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.
Claims
exact text as granted — not AI-modified1 - 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.Join the waitlist — get patent alerts
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