Claims for Patent: 10,781,451

Summary for Patent: 10,781,451

Title:	Antisense oligonucleotides for inducing exon skipping and methods of use thereof
Abstract:	An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.
Inventor(s):	Wilton; Stephen Donald (Applecross, AU), Fletcher; Sue (Bayswater, AU), McClorey; Graham (Bayswater, AU)
Assignee:	The University of Western Australia (Crawley, AU)
Application Number:	16/458,929
Patent Claims:	1. An antisense oligonucleotide of 30 bases comprising the base sequence CUCCAACAUC AAGGAAGAUG GCAUUUCUAG (SEQ ID NO: 181), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide. 2. The antisense oligonucleotide of claim 1, wherein the oligonucleotide is chemically linked to a cholesterol moiety, cholic acid, a thioether, a thiocholesterol, an aliphatic chain, a phospholipid, a polyamine chain, a polyethylene glycol chain, adamantane acetic acid, a palmityl moiety, an octadecylamine moiety, or a hexylamino-carbonyl-oxycholesterol moiety. 3. An injectable solution, comprising the antisense oligonucleotide of claim 1 and a pharmaceutically acceptable carrier or diluent, wherein the injectable solution is formulated for intravenous administration. 4. The injectable solution of claim 3, wherein the oligonucleotide is chemically linked to a cholesterol moiety, cholic acid, a thioether, a thiocholesterol, an aliphatic chain, a phospholipid, a polyamine chain, a polyethylene glycol chain, adamantane acetic acid, a palmityl moiety, an octadecylamine moiety, or a hexylamino-carbonyl-oxycholesterol moiety. 5. The injectable solution of claim 3, wherein the pharmaceutically acceptable carrier or diluent comprises an isotonic saline solution. 6. The injectable solution of claim 5, wherein the isotonic saline solution is phosphate-buffered saline. 7. An injectable solution, comprising the antisense oligonucleotide of claim 1 and a pharmaceutically acceptable carrier or diluent, wherein the injectable solution is formulated for parenteral administration. 8. The injectable solution of claim 7, wherein the oligonucleotide is chemically linked to a cholesterol moiety, cholic acid, a thioether, a thiocholesterol, an aliphatic chain, a phospholipid, a polyamine chain, a polyethylene glycol chain, adamantane acetic acid, a palmityl moiety, an octadecylamine moiety, or a hexylamino-carbonyl-oxycholesterol moiety. 9. The injectable solution of claim 7, wherein the pharmaceutically acceptable carrier or diluent comprises an isotonic saline solution. 10. The injectable solution of claim 9, wherein the isotonic saline solution is phosphate-buffered saline. 11. An injectable solution, comprising the antisense oligonucleotide of claim 1 and a pharmaceutically acceptable carrier or diluent, wherein the injectable solution is formulated for intramuscular administration. 12. The injectable solution of claim 11, wherein the oligonucleotide is chemically linked to a cholesterol moiety, cholic acid, a thioether, a thiocholesterol, an aliphatic chain, a phospholipid, a polyamine chain, a polyethylene glycol chain, adamantane acetic acid, a palmityl moiety, an octadecylamine moiety, or a hexylamino-carbonyl-oxycholesterol moiety. 13. The injectable solution of claim 11, wherein the pharmaceutically acceptable carrier or diluent comprises an isotonic saline solution. 14. The injectable solution of claim 13, wherein the isotonic saline solution is phosphate-buffered saline.