Redefining what’s possible in cystic fibrosis treatment
Our vision is to fully normalize CFTR function
Pipeline
We are a clinical-stage company on a mission to develop highly effective and differentiated treatments for cystic fibrosis (CF) to normalize the function of CFTR, the key protein associated with the disease. We are advancing a pipeline of small molecules engineered to correct the defects caused by F508del, the most common CF mutation, which resides in the nucleotide-binding domain 1 (NBD1) of CFTR. Our first-in-class programs directly target NBD1, which we believe is the key mechanism to enable full CFTR correction. We’re also advancing complementary programs that target ICL4 and TMD1, two other important domains of the CFTR protein affecting CF.
We believe this pipeline has the potential to deliver best-in-class options for CF patients and set a new standard of care in CF.
Core Values
Intentional Innovation
Unwavering Integrity
Continuous Collaboration
Flexible First
Active Trust
Every day I get to go to work and interact with an awesome group of people. Even cooler than that, we all do work that could positively impact patients’ lives someday!
Our ability to target NBD1 is truly novel. I am grateful for the opportunity to work toward the goal of normal CFTR function for CF patients.