Claims for Patent: 10,995,337

Summary for Patent: 10,995,337

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 214.
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:	16/881,430
Patent Claims:	1. A method of treating Duchenne muscular dystrophy, comprising administering an effective amount of an antisense oligonucleotide comprising a base sequence 25 bases in length that is 100% complementary to 25 consecutive bases of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide is chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO: 193), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping. 2. The method of claim 1, wherein the antisense oligonucleotide is formulated with a pharmaceutically acceptable carrier or diluent for intravenous administration. 3. The method of claim 2, wherein the pharmaceutically acceptable carrier or diluent comprises an isotonic saline solution. 4. The method of claim 3, wherein the isotonic saline solution is phosphate-buffered saline. 5. An injectable solution, comprising: a) an antisense oligonucleotide comprising a base sequence 25 bases in length that is 100% complementary to 25 consecutive bases of a target region of exon 53 of the human dystrophin pre-mRNA, said morpholino antisense oligonucleotide is chemically linked to a polyethylene glycol chain; wherein the antisense oligonucleotide base sequence comprises at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO: 193), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the target region and induces exon 53 skipping; and b) a pharmaceutically acceptable carrier or diluent, wherein the injectable solution is formulated for parenteral administration. 6. The injectable solution of claim 5, wherein the pharmaceutically acceptable carrier or diluent comprises an isotonic saline solution. 7. The injectable solution of claim 6, wherein the isotonic saline solution is phosphate-buffered saline.