US2022325280A1PendingUtilityA1

Antisense oligonucleotides and their use for treating pendred syndrome

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Assignee: ASOCURA PHARMACEUTICALS SUZHOU CO LTDPriority: Feb 12, 2020Filed: Apr 18, 2022Published: Oct 13, 2022
Est. expiryFeb 12, 2040(~13.5 yrs left)· nominal 20-yr term from priority
Inventors:Yimin Hua
A61P 27/16C12N 2310/3341C12N 2310/315C12N 2310/11C12N 2310/321C12N 15/1138A61K 31/7088C12N 2320/33A61K 48/005C12N 2310/322C12N 15/113
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Claims

Abstract

The present disclosure relates to the field of medicine. In particular, it relates to novel antisense oligonucleotides that prevent or reduce exon 8 skipping in the SLC26A4 gene during pre-mRNA splicing, and their use in the treatment of Pendred Syndrome.

Claims

exact text as granted — not AI-modified
1 . An antisense oligonucleotide of 10-30 nucleotides in length comprising all or a portion of SEQ ID NO:1 (5′-tgtattagtactaagaggaacacca-3′). 
     
     
         2 - 16 . (canceled) 
     
     
         17 . A method of treating hearing loss in a subject in a subject having Pendred syndrome comprising administering a therapeutically effective amount of the antisense oligonucleotide of  claim 1 . 
     
     
         18 - 28 . (canceled)

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