IP Library › Granted Patent US 10,758,629
Granted Patent B2
US 10,758,629 · App. 15/993,267 · Granted Sep 1, 2020

Exon skipping oligomer conjugates for muscular dystrophy

Inventors: Richard K. Bestwick (Cambridge, MA); Frederick Joseph Schnell (Cambridge, MA)
Assignee: Sarepta Therapeutics, Inc.
A61K47/6807A61K9/0019C07K14/4707C12N15/113C12N15/62
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Quick Facts
Patent No.
US 10,758,629
App. No.
15/993,267
Granted
Sep 1, 2020
Kind
B2
Abstract

Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.

Claims (5)

1. An antisense oligomer of Formula (VIIA):

or a pharmaceutically acceptable salt thereof.

2. A pharmaceutical composition, comprising an antisense oligomer of claim 1 , or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.

3. An antisense oligomer of Formula (VILA):

4. A pharmaceutical composition, comprising an antisense oligomer of claim 3 and a pharmaceutically acceptable carrier.

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 Sep 29, 2020
From: BESTWICK, RICHARD K.; SCHNELL, FREDERICK JOSEPH
To: SAREPTA THERAPEUTICS, INC.
Reel/Frame 053919/0364 →