In recent years, efforts have been focused on the development of small molecule CFTR modulators (CF drug development pipeline; http://www.cff.org/trials/pipeline), culminating in the recent FDA approval of Trikafta (Vertex Pharmaceuticals), the combination of the correctors VX-661 (tezacaftor) plus VX-445 (elexacaftor) and the gating potentiator VX-770 (ivacaftor), for treatment of patients with at least 1 F508del allele. Here, CFTR is linked to cystic fibrosis.