US2018273946A1PendingUtilityA1

Modulation of transthyretin expression

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Assignee: IONIS PHARMACEUTICALS INCPriority: Apr 29, 2010Filed: Oct 11, 2017Published: Sep 27, 2018
Est. expiryApr 29, 2030(~3.8 yrs left)· nominal 20-yr term from priority
Inventors:Brett P. MoniaSusan M. FreierAndrew M. SiwkowskiShuling Guo
A61P 7/00A61P 9/00A61P 9/04A61P 9/06A61P 3/10A61P 7/10A61P 7/04A61P 25/02A61P 25/28A61P 25/22A61P 3/00A61P 25/18A61P 25/20A61P 25/08A61P 25/24A61P 27/06A61P 27/02A61K 48/00C07H 21/04C12N 2310/321C12N 2310/14A61P 13/00A61P 15/00A61P 1/12A61K 31/712A61P 19/10C12N 2320/30A61P 25/00C12N 2310/315C12N 2310/341C12N 15/113A61P 1/08C12N 2310/52C12N 2310/11C12N 15/11A61P 11/00A61P 21/00C12N 2310/3341A61P 1/10A61P 15/10A61P 1/16C12N 2310/3521
65
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Claims

Abstract

Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate transthyretin amyloidosis, or a symptom thereof.

Claims

exact text as granted — not AI-modified
1 - 40 . (canceled) 
     
     
         41 . A compound consisting of a conjugate group and a sodium salt of a single-stranded modified oligonucleotide consisting of 20 linked nucleosides having a nucleobase sequence consisting of SEQ ID NO: 80, wherein the modified oligonucleotide comprises:
 a gap segment consisting of ten linked deoxynucleosides;   a 5′ wing segment consisting of five linked nucleosides; and   a 3′ wing segment consisting of five linked nucleosides;   wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment, wherein each nucleoside of each wing segment comprises a 2′-O-methoxyethyl sugar, wherein each internucleoside linkage is a phosphorothioate linkage, and wherein each cytosine of the modified oligonucleotide is a 5-methylcytosine.   
     
     
         42 . A composition comprising the compound of  claim 41 . 
     
     
         43 . A method of treating transthyretin amyloidosis in an individual comprising administering to the individual the compound of  claim 41 . 
     
     
         44 . The method of  claim 43 , wherein the transthyretin amyloidosis is familial amyloid polyneuropathy (FAP). 
     
     
         45 . The method of  claim 43 , wherein the transthyretin amyloidosis is familial amyloid cardiopathy (FAC). 
     
     
         46 . A method of treating transthyretin amyloidosis in an individual comprising administering to the individual the composition of  claim 42 . 
     
     
         47 . The method of  claim 46 , wherein the transthyretin amyloidosis is familial amyloid polyneuropathy (FAP). 
     
     
         48 . The method of  claim 46 , wherein the transthyretin amyloidosis is familial amyloid cardiopathy (FAC).

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