US2022033820A1PendingUtilityA1

Compositions for treating muscular dystrophy

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Assignee: SAREPTA THERAPEUTICS INCPriority: Mar 15, 2013Filed: Oct 21, 2021Published: Feb 3, 2022
Est. expiryMar 15, 2033(~6.6 yrs left)· nominal 20-yr term from priority
Inventors:Edward M. Kaye
C12N 15/113A61K 31/7125A61K 9/0019A61P 1/00C12N 2320/30C12N 2310/33A61P 21/00A61P 21/02
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Claims

Abstract

Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.

Claims

exact text as granted — not AI-modified
1 - 23 . (canceled) 
     
     
         24 . An antisense oligonucleotide of 23 nucleotides in length, comprising the nucleotide sequence of SEQ ID NO: 112, wherein the uracil bases are optionally thymine bases. 
     
     
         25 . The antisense oligonucleotide of  claim 24 , wherein the antisense oligonucleotide is a phosphorodiamidate morpholino oligomer. 
     
     
         26 . The antisense oligonucleotide of  claim 24  or  25 , wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain.

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