In the previous phases (Phases 1, Phase 2, Phase 3, and Phase 4), the project identified several compounds able to correct the defective CFTR-F508del protein by acting as class III correctors in combination with class I correctors. These compounds were tested in vitro on cells and respiratory tissues from people with cystic fibrosis, demonstrating significant improvements in CFTR function.

In Phase 5, a selected set of molecules will be evaluated using advanced functional assays to assess their ability to restore CFTR activity and improve mucociliary transport, a key process for effective lung clearance, under both physiological and disease-relevant conditions. The ultimate aim is to identify the most promising candidate for further development with academic or industrial partners.

In parallel, the SH compound series will be further analyzed to identify chemical modifications that enhance efficacy and safety. Two additional subgroups derived from the PP series, termed FLM and AL, will also be assessed to select compounds with optimal properties.

Overall, the project seeks to advance the development of new CFTR-correcting drugs, providing people with cystic fibrosis with more effective and safer therapeutic options and laying the groundwork for future innovation.