US2018320188A1PendingUtilityA1

Compositions and methods for modulating growth hormone receptor expression

Assignee: IONIS PHARMACEUTICALS INCPriority: May 1, 2014Filed: May 1, 2018Published: Nov 8, 2018
Est. expiryMay 1, 2034(~7.7 yrs left)· nominal 20-yr term from priority
A61P 5/06A61P 43/00A61P 19/00C12N 2310/351C12N 2310/315C12N 2310/3231C12N 2310/3341A61K 47/555C12N 15/1138C12N 2310/346C12N 2310/321A61K 31/7088C12N 2310/11C12N 2310/341C12N 2310/3525A61K 47/56A61K 47/549C12N 15/11C07K 1/00C07H 21/04C12N 5/00
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Claims

Abstract

The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with excess growth hormone using antisense compounds or oligonucleotides targeted to growth hormone receptor (GHR).

Claims

exact text as granted — not AI-modified
1 .- 192 . (canceled) 
     
     
         193 . A compound consisting of a conjugate group and a modified oligonucleotide according to the following formula: 
       
         
           
           
               
               
           
         
       
     
     
         194 . A composition comprising the compound of  claim 193  or salt thereof and at least one of a pharmaceutically acceptable carrier or diluent. 
     
     
         195 . A prodrug comprising the compound of  claim 193 . 
     
     
         196 . (canceled) 
     
     
         197 . (canceled) 
     
     
         198 . A method comprising administering to an animal the compound  claim 193 . 
     
     
         199 . The method of  claim 198 , wherein the animal is a human. 
     
     
         200 . A method of treating a disease associated with excess growth hormone in a human comprising administering to the human a therapeutically effective amount of the compound of  claim 193  thereby treating the disease associated with excess growth hormone. 
     
     
         201 . The method of  claim 200 , wherein the disease associated with excess growth hormone is acromegaly. 
     
     
         202 . The method of  claim 200 , wherein the treatment reduces IGF-1 levels. 
     
     
         203 . A method of preventing a disease associated with excess growth hormone in a human comprising administering to the human a therapeutically effective amount of a compound of  claim 193  thereby preventing the disease associated with excess growth hormone. 
     
     
         204 . The method of  claim 203 , wherein the disease associated with excess growth hormone is acromegaly. 
     
     
         205 . The method of  claim 200 , comprising co-administering the compound and a second agent. 
     
     
         206 . The method of  claim 205 , wherein the compound or composition and the second agent are administered concomitantly. 
     
     
         207 . A method of reducing growth hormone receptor (GHR) levels in a human comprising administering to the human a therapeutically effective amount of the compound of  claim 193 , thereby reducing GHR levels in the human. 
     
     
         208 . The method of  claim 207 , wherein the human has a disease associated with excess growth hormone. 
     
     
         209 . The method of  claim 207 , wherein the disease associated with excess growth hormone is acromegaly. 
     
     
         210 . The method of  claim 209 , comprising co-administering the compound and a second agent. 
     
     
         211 . The method of  claim 210 , wherein the compound and the second agent are administered concomitantly. 
     
     
         212 . (canceled) 
     
     
         213 . (canceled)

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