US2022090084A1PendingUtilityA1

Antisense-induced exon exclusion in myostatin

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Assignee: SAREPTA THERAPEUTICS INCPriority: Mar 18, 2015Filed: Apr 15, 2021Published: Mar 24, 2022
Est. expiryMar 18, 2035(~8.7 yrs left)· nominal 20-yr term from priority
Inventors:Frederick Joseph SchnellRichard K. Bestwick
A61P 21/00C12N 2310/3233C12N 15/1136C07H 21/00C07D 241/02C12N 2310/3513C07D 265/28C12N 2310/11C12N 2310/3145C12N 2320/33C12N 2310/346A61K 31/7105
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Claims

Abstract

The present disclosure relates to antisense oligomers and related compositions and methods for decreasing the expression of functional human myostatin and methods for treating muscular dystrophy and related disorders and more specifically relates to inducing exclusion of myostatin exon 2 and thereby reducing levels of myostatin protein.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . An antisense oligomer compound comprising a sequence selected from SEQ ID NOS: 2-5, 10-15, and 17-30, wherein X is independently selected from uracil (U) or thymine (T), comprising: at least one subunit that is a nucleotide analog having (i) a modified internucleoside linkage (ii) a modified sugar moiety, or (iii) a combination of the foregoing.

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