IP Library › Granted Patent US 9,217,148
Granted Patent B2
US 9,217,148 · App. 14/213,607 · Granted Dec 22, 2015

Exon skipping compositions for treating muscular dystrophy

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Quick Facts
Patent No.
US 9,217,148
App. No.
14/213,607
Granted
Dec 22, 2015
Kind
B2
Abstract

Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.

Claims (6)

1. An antisense oligonucleotide of 28 bases comprising the base sequence GAAACTGTTCAGCTTCTGTTAGCCACTG (SEQ ID NO: 6), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide.

2. The antisense oligonucleotide of claim 1 , 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.

3. The antisense oligonucleotide of claim 1 , wherein the oligonucleotide is chemically linked to a polyethylene glycol moiety.

4. A pharmaceutical composition comprising an antisense oligonucleotide of 28 bases comprising the base sequence GAAACTGTTCAGCTTCTGTTAGCCACTG (SEQ ID NO: 6), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and a pharmaceutically acceptable carrier.

5. The pharmaceutical composition of claim 4 , 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.

6. The pharmaceutical composition of claim 4 , wherein the oligonucleotide is chemically linked to a polyethylene glycol moiety.

Assignments (2)
SECURITY INTEREST Recorded May 7, 2025
From: SAREPTA THERAPEUTICS, INC.
To: JPMORGAN CHASE BANK, N.A. AS ADMINISTRATIVE AGENT
Reel/Frame 071218/0445 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 1, 2014
From: BESTWICK, RICHARD K; FRANK, DIANE ELIZABETH
To: SAREPTA THERAPEUTICS, INC.
Reel/Frame 033443/0264 →