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US 2010/0249233 A1Published application
Compound and use thereof in the treatment of amyloidosis
AssigneePentraxin Therapeutics Limited
InventorsMark Brian PEPYS, Steven Victor Ley, Angus John Morrison, Vittorio Bellotti, Stephen Wood, Simon Kolstoe, Martin Smith
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Abstract
The present invention relates to compounds of formula (I) for stabilizing the tetrameric form of transthyretin, compounds for use in the treatment or prevention of amyloidosis, and agents and medicaments comprising such compounds. wherein X, Y, R1, R2, R3, R4, m, n, p, q, and the linker are as defined herein.
Claims19
Agent for stabilising the tetrameric form of transthyretin, which comprises a compound of the general formula (I) or a pharmaceutically acceptable salt, ester or prodrug thereof:
- wherein:
- each Y is independently a direct bond or —CH2—;
- each X is independently —NH—, —O—, —S—, —CH2—, —NR—, —CO—, —CONH—, —CONR—, —C═N—O—, —NHCO—, —NRCO—, —O—N═C—, —SO—, —SO2— or a direct bond, wherein
- each of R1, R2, R3 and R4 is independently F, Cl, Br, I, CF3, OCF3, R′, OR′, NR′R′, SOR′ or SO2R′,
- wherein R and R′ are each independently C1-C3alkyl which is straight or branched chain or cyclic optionally substituted by one or more halogen atoms;
- and each m, n, p and q is independently 0 to 4, wherein m+n+p+q>0;
- and wherein the linker is a linear or branched chain of 7 to 13 carbon atoms in which one or more of the carbon atoms are optionally replaced by a heteroatom, wherein the said chain is unsubstituted or substituted by one or more groups comprising halogen, O, or N atoms, or OH, C1-C3 alkyl, C2-C3 alkenyl, C2-C3 alkynyl or C1-C3 alkoxy.
4 more elements
Dependencies are inferred from the claim text.