Exon skipping oligomer conjugates for muscular dystrophy
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
1. An antisense oligomer conjugate of Formula (IV):
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 conjugate is of Formula (IVA):
4. A pharmaceutical composition, comprising an antisense oligomer conjugate of Formula (IV):
or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.
5. The pharmaceutical composition of claim 4 , wherein the antisense oligomer conjugate is in the form of a pharmaceutically acceptable salt.
6. A pharmaceutical composition comprising an antisense oligomer conjugate of Formula (IVA):
and a pharmaceutically acceptable carrier.