About us
What we do

We are developing CFTR modulator combinations to provide more drug options for people with CF. Our drug candidates address the synthesis, folding, trafficking, and function of the CFTR protein.

We completed a global Phase 2 program

for people with one or two copies of F508del  to investigate our double and triple combinations. Our triple combination includes posenacaftor, a corrector, dirocaftor, a potentiator, and nesolicaftor, an amplifier. The results of the study support further development of our triple combination in patients who are homozygous for the F508del mutation.

We are participating in the HIT-CF Europe project

to address the high unmet needs of people with CF who have rare mutations. Through this collaboration, we are helping to pave the way for personalized medicine in cystic fibrosis. Our drug candidates are being investigated for potential benefit in less common mutations for which there are no approved modulator treatments.