Exon skipping oligomer conjugates for muscular dystrophy
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.
1. An antisense oligomer conjugate of Formula (III):
or a pharmaceutically acceptable salt thereof.
2. A pharmaceutically acceptable salt of the antisense oligomer conjugate of claim 1 .
3. The antisense oligomer conjugate of claim 1 , wherein the antisense oligomer is of Formula (IIIA):
4. The antisense oligomer conjugate of claim 1 , wherein the antisense oligomer is of Formula (IV):
or a pharmaceutically acceptable salt thereof.
5. A pharmaceutically acceptable salt of the antisense oligomer conjugate of claim 4 .
6. The antisense oligomer conjugate of claim 4 , wherein the antisense oligomer is of Formula (IVA):
7. A pharmaceutical composition comprising the antisense oligomer conjugate of claim 1 , or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.
8. A pharmaceutical composition comprising the antisense oligomer conjugate of claim 3 and a pharmaceutically acceptable carrier.
9. A pharmaceutical composition comprising the antisense oligomer conjugate of claim 4 and a pharmaceutically acceptable carrier.
10. A pharmaceutical composition comprising the antisense oligomer conjugate of claim 6 and a pharmaceutically acceptable carrier.