22 results about "Cftr modulator" patented technology

Crystalline forms of salts of Compound I are disclosed. Pharmaceutical compositions comprising the same, methods of treating cystic fibrosis using the same, and methods for making the same are also disclosed.

The present invention concerns the compounds of formula (I) wherein R1, R2, R3, R4, X, Ar1 and Ar2 are as described in the description, and their use in the treatment of CFTR-related diseases and disorders, especially in the treatment of cystic fibrosis, in combination with one or more therapeutically active ingredients acting as CFTR modulator(s), wherein said CFTR modulator(s) is / are CFTR one or more CFTR corrector(s) and / or a CFTR potentiator. The invention further relates to pharmaceutical compositions comprising the compounds of formula (I) in combination with one or more therapeutically active ingredients acting as CFTR modulator(s), wherein said CFTR modulator(s) is / are one or more CFTR corrector(s) and / or a CFTR potentiator.

The invention relates to crystalline form of (3S,7S,10R,13R)-13-benzyl-20-fluoro-7-isobutyl-N-(2-(3-methoxy-1,2,4-oxadiazol-5-yl)ethyl)-6,9-dimethyl-1, 5,8,11-tetraoxo-10-(2,2,2-trifluoroethyl)-1,2,3,4,5,6,7,8,9,10,11,12,13,14-tetradecahydro-[1]oxa[4,7,10,14]tetraazacycloheptadecino[16,17-f]quinoline-3-carboxamide; processes for the preparation thereof, pharmaceutical compositions containing such crystalline form, pharmaceutical compositions prepared from such crystalline form, and their use as a medicament, especially as CFTR modulators.

Disclosed herein are pharmaceutical compositions for the treatment of respiratory diseases and disorders, including rhinosinusitis. In some aspects, the compositions provide selective delivery to the upper airway and facilitate the transport active agents effectively across membranes.

Disclosed herein are pharmaceutical compositions for the treatment of respiratory diseases and disorders, including rhinosinusitis. In some aspects, the compositions provide selective delivery to the upper airway and facilitate the transport active agents effectively across membranes.

Processes and methods of preparing Compound I are disclosed. Crystalline forms of Compound I, pharmaceutically acceptable salts, solvates, hydrates, and cocrystals thereof, pharmaceutical compositions comprising the same, methods of treating cystic fibrosis using the same, and methods for making the same are also disclosed.

This disclosure relates to compounds that are cystic fibrosis transmembrane conductance regulator (CFTR) modulators and pharmaceutical compositions containing the same. In certain embodiments, this disclosure relates to methods of managing a CFTR related disease or condition or respiratory distress comprising administering an effective amount of a CFTR modulator disclosed herein to a subject in need thereof.

The present application relates to the new use of CFTR modulators as the treatment of IgG4-related diseases, belonging to the field of biopharmacy. Specifically, the present application finds that CFTR modulators can significantly reduce the concentration of IgG1 in serum and reduce the inflammatory characteristics of the kidney in IgG4-related disease model mice through non-clinical pharmacodynamic tests, and has good therapeutic effect on IgG4-related diseases, and has the new use as the treatment of IgG4-related diseases. The present application also relates to a pharmaceutical composition comprising a CFTR modulator drug and a combination for such use.

The invention discloses a synthesis method of a cis-1, 4-difluoromethyl and amido substituted cycloalkene compound, which is characterized by comprising the following steps: reacting halide as shown in a formula II structure, 1, 3-cyclodiene as shown in a formula III structure and a nitrogen nucleophilic reagent as shown in a formula IV structure under the action of tetrakis (triphenylphosphine) palladium, blue light and an acid-binding agent to obtain the cis-1, 4-difluoromethyl and amido substituted cycloalkene compound as shown in a formula I structure. The invention discloses a 1, 4-difluoromethyl and amido substituted cycloalkene compound. The method has the advantages of easily available reaction raw materials, mild reaction conditions, simple operation, wide substrate application range, good functional group compatibility and good application prospect. And when the reaction is applied to synthesis of CFTR modulator, a target product can be obtained by only four steps.

Crystalline forms of Compound I:pharmaceutically acceptable salts thereof, and solvates and hydrates thereof are disclosed. Pharmaceutical compositions comprising the same, methods of treating cystic fibrosis using the same, and methods for making the same are also disclosed.

The invention therefore provides methods of treating a respiratory disease characterized by mucus hyper-secretion comprising administering to a human patient in need of such treatment a gamma secretase inhibitor (GSI), wherein the administration of a GSI is effective in reducing mucus in such patient's lungs or inhibiting mucus accumulation in said patient's lungs. In some embodiments, the methods of the invention are effective in treating a respiratory disease selected from the group consisting of cystic fibrosis, chronic obstructive pulmonary disease, primary ciliary dyskinesis, chronic bronchitis, asthma, idiopathic and secondary bronchiectasis, bronchiolitis obliterans, idiopathic pulmonary fibrosis, respiratory infection and mucus accumulation in response to infection. Methods of the invention further include methods of treating cystic fibrosis wherein a GSI is administered to a patient being administered or in need of a CFTR modulator, wherein the mucus in such patient's lungs is reduced or mucus accumulation in such patient's lungs is inhibited.

Provided herein are pharmaceutical combinations comprising recombinant adeno-associated virus (AAV) and CFTR modulator(s) for the treatment of cystic fibrosis.

This disclosure relates to compounds that are cystic fibrosis transmembrane conductance regulator (CFTR) modulators and pharmaceutical compositions containing the same. In certain embodiments, this disclosure relates to methods of managing a CFTR related disease or condition or respiratory distress comprising administering an effective amount of a CFTR modulator disclosed herein to a subject in need thereof.