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US 7,683,082 B2Granted
1,2,4-oxadiazole benzoic acid compositions and their use in bioassays
AssigneePTC Therapeutics, Inc.
InventorsGary Mitchell Karp, Seongwoo Hwang, Guangming Chen, Neil Gregory Almstead, Young-Choon Moon
Life of this patent
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Abstract
Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.
Claims4
A method for testing whether a compound increases activity of a protein encoded by an mRNA that contains a premature stop codon, comprising:
- (a) contacting a composition comprising a cell extract or cell lysate and the mRNA with an effective amount of a compound of the formula:
- or a pharmaceutically acceptable salt thereof, wherein:
- Z is substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted cycloalkyl, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted heterocycle, substituted or unsubstituted arylalkyl;
- R1 is hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, —(CH2CH2O)nR6 or any biohydrolyzable group;
- R2, R3, R4, R5 and R6 are independently hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl;
- substituted or unsubstituted cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, alkoxy, aryloxy, heteroaryloxy, halogen, CF3, OCF3, OCHF2, CN, COOH, COOR7, SO2R7, NO2, NH2, or N(R7)2;
- each occurrence of R7 is independently hydrogen. substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl;
- substituted or unsubstituted cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, alkoxy, aryloxy, heteroaryloxy, halogen or CF3;
- and
- n is an integer from 1 to 7;
- and
- (b) detecting the activity of the protein, wherein a compound that increases the activity of the protein is detected if there is an increase in the activity of the protein in the presence of the compound relative to the absence of the compound.
9 more elements
Dependencies are inferred from the claim text.