Granted Patent
B2
US 10,765,760 · App. 15/993,287 · Granted Sep 8, 2020
Exon skipping oligomer conjugates for muscular dystrophy
Inventors:
Gunnar J. Hanson (Cambridge, MA); Marco A. Passini (Cambridge, MA); Frederick Joseph Schnell (Cambridge, MA)
Assignee:
Sarepta Therapeutics, Inc.
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Abstract
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.
Claims (5)
1. An antisense oligomer conjugate of Formula (IV):
or a pharmaceutically acceptable salt thereof.
2. The antisense oligomer conjugate of claim 1 , wherein the antisense oligomer is of Formula (IVA):
3. A pharmaceutical composition, comprising an antisense oligomer conjugate of claim 1 , or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.
4. A pharmaceutical composition, comprising an antisense oligomer conjugate of claim 2 , or a pharmaceutically acceptable salt thereof, 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 Aug 5, 2022
From: HANSON, GUNNAR J.; PASSINI, MARCO A.; SCHNELL, FREDERICK JOSEPH
To: SAREPTA THERAPEUTICS, INC.
Reel/Frame 060737/0238 →