US2012238632A1PendingUtilityA1

Modulators of retinol-retinol binding protein (rbp)-transthyretin (ttr) complex formation

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Individually held — no corporate assignee on recordPriority: Nov 4, 2004Filed: Sep 13, 2011Published: Sep 20, 2012
Est. expiryNov 4, 2024(expired)· nominal 20-yr term from priority
Inventors:Nathan L. MataYun HanKenneth J. WidderJay Lichter
A61P 27/02A61P 27/00G01N 2500/02A61K 41/0061G01N 33/6893A61K 31/203A61K 31/215A61P 21/04A61P 17/00A61K 31/16A61K 49/0052A61K 49/0056G01N 2800/164A61K 49/0041G01N 33/542G01N 33/53
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Claims

Abstract

Described herein are methods and compositions for the detection of transthyretin (TTR), retinol binding protein (RBP) and retinol complex formation. The methods and compositions described herein also provide for the screening of modulators of retinol-RBP-TTR complex formation. Furthermore, the methods and compositions provide for therapeutic agents for the treatment and/or prevention of age-related macular degeneration and/or dystrophies.

Claims

exact text as granted — not AI-modified
1 . A method for treating Stargardt disease in a human in need comprising systemic administration of a therapeutically effective amount of a compound having the structure of Formula (I): 
       
         
           
           
               
               
           
         
         wherein X 1  is NR 2 ; R 1  is (CHR 2 ) x -L 1 -R 3 , wherein x is 0; L 1  is a single bond; each R 2  is H; and R 3  is a mono-substituted aryl wherein said mono substituted aryl is 4-hydroxyphenyl or 4-methoxyphenyl. 
       
     
     
         2 . The method of  claim 1 , wherein said mono-substituted aryl is 4-hydroxyphenyl. 
     
     
         3 . The method of  claim 1 , wherein said mono-substituted aryl is 4-methoxyphenyl. 
     
     
         4 . The method of  claim 1 , wherein said human is a carrier of mutant ABCA4 or ELOV4 gene.

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