Organoid-Guided drug trial offers hope for cystic fibrosis patients with rare mutations
Disease control
Completed
This study tested a triple-drug combination (dirocaftor/posenacaftor/nesolicaftor) in 41 adults with cystic fibrosis who have rare CFTR gene mutations not covered by existing treatments. Participants were chosen based on lab tests using their own intestinal organoids to predict w…
Phase: PHASE2 • Sponsor: Kors van der Ent • Aim: Disease control
Last updated Jun 27, 2026 07:59 UTC