Background The most frequent mutation among patients with cystic fibrosis (CF), F508del, causes defective maturation and early degradation of CFTR protein1. The F508del defect can be targeted with compounds known as correctors2.  Proteins carrying F508del and other mutations also show a channel gating defect that can be addressed with another type of compounds called potentiators3.  Currently, there are no F508del-CFTR correctors with adequate efficacy in human. Therefore, new compounds are needed4.

Hypothesis and objectives The project aims at the selection of a compound to progress to preclinical development, and at starting preclinical development activities. In parallel, activities to identify a backup compound will be conducted.