Modulators of retinol-retinol binding protein (rbp)-transthyretin (ttr) complex formation
AssigneeReVision Therapeutics, Inc.
InventorsNathan L. Mata, Yun Han, Kenneth Widder, Jay Lichter
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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.
Claims4
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 X1 is NR2;
- R1 is (CHR2)x-L1-R3, wherein x is 0;
- L1 is a single bond;
- each R2 is H;
- and R3 is a mono-substituted aryl wherein said mono substituted aryl is 4-hydroxyphenyl or 4-methoxyphenyl.
Dependencies are inferred from the claim text.