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US 2022/0017901 A1Published application
Exon skipping oligomer conjugates for muscular dystrophy
AssigneeSarepta Therapeutics, Inc.
InventorsFrederick Joseph Schnell, Baozhong Cai, Ankur Desai, Richard K. Bestwick
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Abstract
Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 50 skipping are described. In various aspects, antisense oligomers are described according to Formula (I): or a pharmaceutically acceptable salt thereof, wherein T, Nu, n, and R100 are defined herein.
Claims10
An antisense oligomer according to Formula (I):
- or a pharmaceutically acceptable salt thereof, wherein:
- each Nu is a nucleobase which taken together form a targeting sequence;
- T is a moiety selected from:
- and the distal —OH or —NH2 of the T moiety is optionally linked to a cell-penetrating peptide;
- R100 is hydrogen or a cell-penetrating peptide;
- each Nu from 1 to n and 5′ to 3′ corresponds to the nucleobases:
- wherein A is
- C is
- G is
- and T is
7 more elements
-8. (canceled)
-17. (canceled)
Dependencies are inferred from the claim text.