Exon skipping oligomer conjugates for muscular dystrophy
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 45 skipping are described.
1. An antisense oligomer conjugate of Formula (IV):
(peptide is SEQ ID NO: 4) or a pharmaceutically acceptable salt thereof.
2. The antisense oligomer conjugate of claim 1 , wherein the antisense oligomer is of Formula (IVA):
(IVA) (peptide is SEQ ID NO:4).
3. A pharmaceutical composition, comprising an antisense oligomer conjugate of claim 1 , or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.
4. The antisense oligomer conjugate of claim 1 , wherein the antisense oligomer conjugate is a pharmaceutically acceptable salt of Formula (IV).
5. A pharmaceutical composition, comprising an antisense oligomer conjugate of claim 2 , or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.
6. An antisense oligomer conjugate of Formula (I):
or a pharmaceutically acceptable salt thereof, wherein:
each Nu is a nucleobase which taken together form a targeting sequence having SEQ ID NO: 1 (5′-CAATGCCATCCTGGAGTTCCTG-3′); and
T is
7. A pharmaceutical composition, comprising an antisense oligomer conjugate of claim 6 , or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.