Top Sector Life Sciences & Health (LSH) entails a broad scope of disciplines, from pharmaceuticals to medical technology and from healthcare infrastructure to vaccination. To realise its mission – vital citizens in a healthy economy - the Top Sector builds on the strengths of the Dutch LSH sector to address the biggest societal challenges in prevention, cure and care. By funding multidisciplinary public-private partnerships (PPPs) the Top Sector aims to facilitate innovation. Here we give an overview of  a number of funded R&D projects by Top Sector LSH. The page is updated continuously.

Mode of action of candidate drugs for treatment of Cystic Fibrosis

Disease-modifying drug discovery for the treatment of Cystic Fibrosis including translational aspects to patients.

Cystic Fibrosis (CF) is a genetic disease (more than  100.000 patients worldwide) and is caused by a defect in a protein (CFTR). Each protein in our body is a chain of amino acids that must be properly folded in the cell to perform optimally. That folding of the CFTR protein is a complicated process. In many patients with CF this folding process is impaired and therefore an effective protein cannot be produced. This is disrupting the salt and water resources in all cells that produce mucus and hence tough slime is created.

Recent research results show that the basic defect in the cells in CF can be tackled with medicines. Because the current medications are insufficiently working or only working for a group of patients with specific abnormalities in the genetic material (mutations), Galapagos is one of the companies developing candidate medicines to target these basic defects.

Researchers from Utrecht University Department of Chemistry,  Pharmaceutical Company Galapagos and the  Nederlandse Cystic Fibrosis Stichting  (NCFS) have jointly performed a project to better understand  the disease mechanisms  of Cystic Fibrosis and  the mode of action of new candidate drugs being developed by Galapagos, in order to bring better drugs faster to patients with CF. By using the techniques that have been developed by the Lab of Professor Ineke Braakman to study the mode of action of the Galapagos candidate drugs, this project has gained much more  insights into the basic defect in the cell at different mutations, how candidate medicines of Galapagos are acting thereon and how the cells and auxiliary proteins respond to them.

These results shall be used in the further development of the best candidate medicines by Galapagos and other companies. In already granted follow-up projects the same consortium partners will further unravel the mechanism of the folding of the protein and how to improve this in order to contribute to the ultimate objective: a cure for people with CF.

Websites: Galapagos, Universiteit Utrecht, NCFS