Mechelen-bovelingen, Belgium
Mechelen-bovelingen, Belgium

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The present invention provides for compounds of formula (I) wherein R^(1), R^(2), R^(3), and R^(4 )have any of the values defined in the specification, and pharmaceutically acceptable salts thereof, that are useful as agents in the treatment of diseases and conditions mediated and modulated by CFTR, including cystic fibrosis, Sjgrens syndrome, pancreatic insufficiency, chronic obstructive lung disease, and chronic obstructive airway disease. Also provided are pharmaceutical compositions comprised of one or more compounds of formula (I).


A combination therapy including a modulator of the function (potentiator) of cystic fibrosis transmembrane conductance regulator (CFTR) protein, and one or two modulator(s) of the cellular processing and/or localization molecule (correctors) is provided in a method for treating cystic fibrosis in a subject having a mutation located between the amino acid residues 1164-1480 of full length wild-type CFTR.


Patent
Galapagos | Date: 2016-10-07

A corrector agent capable of stabilizing a newly synthesized cystic fibrosis transmembrane conductance regulator (CFTR) protein, useful in the treatment of cystic fibrosis.


The present invention provides for compounds of formula (I) wherein R^(1), R^(2), R^(3), R^(4), R^(5), and R^(6 )have any of the values defined in the specification, and pharmaceutically acceptable salts thereof, that are useful as agents in the treatment of diseases and conditions mediated and modulated by CFTR, including cystic fibrosis, Sjgrens syndrome, pancreatic insufficiency, chronic obstructive lung disease, and chronic obstructive airway disease. Also provided are pharmaceutical compositions comprised of one or more compounds of formula (I).


The present invention discloses compounds according to Formula I: wherein R^(1), R^(2), R^(3), L, and the subscript m are as defined herein. The present invention relates to compounds and their use in the treatment of cystic fibrosis, methods for their production, pharmaceutical compositions, and methods of treatment using the same, for the treatment of cystic fibrosis by administering a compound of the invention.


The present invention discloses compounds according to Formula I: wherein R^(1 )is as defined herein. The present invention relates to compounds and their use in the treatment of cystic fibrosis, methods for their production, pharmaceutical compositions comprising the same, and methods of treatment cystic fibrosis by administering a compound of the invention.


Patent
Galapagos | Date: 2016-05-25

The invention discloses compounds of Formula I wherein X, R^(1), R^(2), and R^(3 )are as defined herein. The present invention relates to compounds and their use in the treatment of cystic fibrosis, methods for their production, pharmaceutical compositions comprising the same, and methods of treating cystic fibrosis by administering a compound of the invention.


Patent
Galapagos | Date: 2016-07-08

The present invention provides for compounds of formula (I) wherein X, Y, and R^(1 )have any of the values defined in the specification, and pharmaceutically acceptable salts thereof, that are useful as agents in the treatment of diseases and conditions mediated and modulated by CFTR, including cystic fibrosis, Sjgrens syndrome, pancreatic insufficiency, chronic obstructive lung disease, and chronic obstructive airway disease. Also provided are pharmaceutical compositions comprised of one or more compounds of formula (I).


Patent
Galapagos | Date: 2016-07-13

The invention relates to the field of medicinal research, cartilage physiology and diseases involving the degeneration of cartilage tissue. More specifically, the invention relates to methods and means for identifying compounds that inhibit catabolic processes in chondrocytes and that decrease the degradation of cartilage and/or ECM. The invention also relates to the compounds that are useful in the treatment of osteoarthritis. The invention also relates to targets, the modulation of which results in a decrease in the degradation of ECM and /or cartilage and decrease inflammation. In addition, the invention relates to compositions and methods for the use thereof in treating conditions that are characterized by the degradation of ECM and /or cartilage and inflammation.


Grant
Agency: European Commission | Branch: H2020 | Program: MSCA-ITN-EID | Phase: MSCA-ITN-2015-EID | Award Amount: 1.06M | Year: 2016

MIMIC is an interdisciplinary European Industrial Doctorate at the interface of cell biology, engineering and drug development. MIMIC aims to develop and improve novel organs on chips technology. This technology combines modern cell biology with microfluidics and chip-based techniques with the goal to mimic organ functionality. There is a high demand by the pharmaceutical industry for more reliable tissue models to test drug toxicity and drug efficiency at early stages of drug development. Early reliable drug testing will have a major impact on drug development costs and human health. Furthermore, ethical considerations urge for the search for alternatives to replace animal tests in drug development and basic research. Organs on chips are a new exciting possibility to closer mimic human organ functionality in vitro than conventional 2D or 3D cell cultures. Organs on chips allow both, the emulation of healthy organs as well as the emulation of specific disease conditions using corresponding engineered or patient derived human cells. Moreover, organs on chips are ideally suited for high-throughput drug screening. The EID-MIMIC will develop novel organs on chips prototypes, and validate their suitability for end-users for high throughput drug screening or basic research. MIMIC will train early stage researchers in cutting edge technologies, like novel chip based technologies e.g. cell micropatterning, soft-lithography and microfluidics technology, as well as state of the art microscopy like super resolution- and confocal spinning disc microscopy and modern genome editing techniques like CRISPR-technology. In addition, MIMIC has developed a 3 year modular curriculum including workshops on creativity and business skills, summer schools, business plan competitions and international conferences with a specific agenda of transferable skill training elements highly relevant for scientific communication, translational research and, in particular, entrepreneurship.

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