Antisense oligonucleotides for inducing exon skipping and methods of use thereof

Patent metadata

Field	Value
Publication number	US-2018163205-A1
Application number	US-201715673019-A
Country	US
Kind code	A1
Filing date	Aug 9, 2017
Priority date	Jun 28, 2004
Publication date	Jun 14, 2018
Grant date	—

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.

First claim

We claim:

1 . 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.

2 . An antisense molecule according to claim 1 capable of inducing exon skipping in exons 3, 4, 8, 10 to 16, 19 to 40, 42 to 44, 46, 47 and 50 to 53 of the dystrophin gene.

3 . A combination of two or more antisense molecules according to claim 1 or 2 capable of binding to a selected target to induce exon skipping in the dystrophin gene.

4 . A combination or two or more antisense molecules according to claim 3 selected from Table 1B.

5 . A combination of two or more antisense molecules according to claim 1 or 2 joined together to form a “weasel”, wherein said weasel is capable of binding to a selected target to induce exon skipping in the dystrophin gene.

6 . A combination of two or more antisense molecules according to claim 5 selected from Table 1C.

7 . The antisense molecule according to any one of claims 1 to 6 , capable of binding to a selected target site, wherein the target site is an rnRNA splicing site selected from a splicer donor site, splice acceptor sites or exonic splicing enhancer elements.

8 . A method of treating muscular dystrophy in a patient comprising administering to the patient a composition comprising an antisense molecule according to anyone of claims 1 to 6 .

9 . A pharmaceutical or therapeutic composition for the treatment of muscular dystrophy in a patient comprising (a) at least an antisense molecule according to any one of claims 1 to 6 , and (b) one or more pharmaceutically acceptable carriers and/or diluents.

10 . The composition according to claim 9 , comprising about 20 nM to 600 nM of the antisense molecule.

11 . The use of an antisense molecule according to any one of claims 1 to 6 for the manufacture of a medicament for modulation of muscular dystrophy.

12 . An antisense molecule according to any one of claims 1 to 6 for use in antisense molecule based therapy.

13 . An antisense molecule according to any one of claims 1 to 6 as herein before described with reference to the examples.

14 . A kit comprising at least one antisense molecule according to any one of claims 1 to 6 , a suitable carrier and instructions for its use.

Assignees

• Univ Western Australia

Inventors

• Wilton Stephen Donald
• Fletcher Sue
• Mcclorey Graham

Classifications

• A61P21/00

  Drugs for disorders of the muscular or neuromuscular system · CPC title

• C12N15/113 Primary

  Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)} · CPC title

• C12N2310/3341

  5-Methylcytosine · CPC title

• C12N2310/11

  Antisense · CPC title

• C12N2310/315

  Phosphorothioates · CPC title