IP Library › Granted Patent US 12,709,749
Granted Patent B2
US 12,709,749 · App. 17/817,588 · Granted Aug 18, 2026

Antisense molecules and methods for treating pathologies

Inventors: Stephen Donald Wilton (Applecross, AU); Sue Fletcher (Bayswater, AU); Abbie Adams (Kalamunda, AU); Penny Meloni (Mount Hawthorn, AU)
Assignee: The University of Western Australia
C12N15/113C12N15/111C12N2310/11C12N2310/315C12N2310/3181C12N2310/321C12N2310/3233C12N2310/351C12N2320/33
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Quick Facts
Patent No.
US 12,709,749
App. No.
17/817,588
Filed
Aug 4, 2022
Granted
Aug 18, 2026
Kind
B2
Art Unit
1636
USPC
514/44A
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 59.

Claims (6)

1 . An antisense oligonucleotide of 27 bases in length 100% complementary to a target region of exon 44 of the human dystrophin pre-mRNA, wherein the target region is annealing site H44A(+59+85), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 44 skipping;

or a pharmaceutically acceptable salt thereof.

2 . An antisense oligonucleotide of 27 bases comprising the base sequence CUG UUC AGC UUC UGU UAG CCA CUG AUU (SEQ ID NO: 54), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide and is uniformly modified to comprise a 5-substituted pyrimidine base;

or a pharmaceutically acceptable salt thereof.

3 . The antisense oligonucleotide of claim 2 , wherein the antisense oligonucleotide is chemically linked to one or more moieties or conjugates that enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotide.

4 . The antisense oligonucleotide of claim 2 , wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 24, 2025
From: WILTON, STEPHEN DONALD; FLETCHER, SUE; ADAMS, ABBIE; MELONI, PENNY
To: THE UNIVERSITY OF WESTERN AUSTRALIA
Reel/Frame 070307/0726 →
Priority Claims (1)
AU 2009905549 · Nov 12, 2009 · national
Continuity (7)
Continuation 16993116 · Aug 13, 2020
Continuation 16357918 · Mar 19, 2019
Division 15661750 · Jul 27, 2017
Continuation 14944886 · Nov 18, 2015
Continuation 14108137 · Dec 16, 2013
Continuation 13509331 · Nov 12, 2010
Related Publication 20230063394A1 · Mar 2, 2023
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