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US 2016/0095869 A1Published application
Compounds as modulators of a mutant cftr protein and their use for treating diseases associated with cftr protein malfunction
AssigneeInstytut Biochemii I Biofizyki Pan
InventorsNorbert Odolczyk, Piotr ZIELENKIEWICZ, Grzegorz WIECZOREK, Aleksander EDELMAN, Danielle TONDELIER, Janine FRITSCH
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Abstract
An exemplary embodiment relates to novel protein modulators capable of altering function of the mutant CFTR protein and their use for treating diseases associated with CFTR protein malfunction. An exemplary embodiment provides compositions, pharmaceutical preparations and methods of correcting the cellular alteration of a mutant CFTR protein wherein the CFTR mutation is a mutation ΔF508-CFTR, or another mutation of class II.
Claims15
A composition comprising:
- a modulator of a mutant CFTR protein adapted for use in the manufacture of a medication for the treatment of diseases associated with CFTR protein malfunction including:
- a compound of general formula (I)
- its tautomers, E and Z geometrical isomers, optically active forms such as enantiomers, diastereomers and their racemate forms or a mixture of stereoisomeric forms or its pharmaceutically acceptable salts thereof or complexes thereof;
- wherein Z1 is independently selected from the group consisting of:
- —CnH(2n)—, which is branched or unbranched wherein n is an integer from 1 to 5;
- —CnH(2n−2)— in E or Z geometrical conformation which is branched or unbranched wherein n is an integer from 2 to 5;
- —CnH(2n−4)— which is branched or unbranched wherein n is an integer from 2 to 5;
- —CR′H—, —C2H3R′—, E or Z—C2HR′—, —C3H5R′—, E or Z—C3H3R′—, —OCH2—, —CH2O—, —NR″CH2—, —CH2NR″—;
- wherein R′ is independently selected from the group consisting of:
- —H, halogen, —NH2, —OH, —CN, CF3, —CHF2, —CH2F, —SH, —SCN, —CH3, —C2H5;
- wherein R″ is independently selected from the group consisting of:
- —H, —CH3, —C2H5;
- wherein R1 and R2 are independently selected from the group consisting of aromatic ring or heteroaromatic ring.
10 more elements
Dependencies are inferred from the claim text.