Partial rescue of F508del‐cystic fibrosis transmembrane conductance regulator channel gating with modest improvement of protein processing, but not stability, by a dual‐acting small molecule

DOI:

10.1111/bph.14141

Authors:

Jia Liu, Hermann Bihler, Carlos M. Farinha, Nikhil T Awatade, Ana M. Romão, Dayna L. Mercadante, Yi Cheng, Isaac Musisi, Walailak Jantarajit, Yi‐Ting Wang, Zhiwei Cai, Margarida D. Amaral, Martin Mense, David N. Sheppard

Abstract:

CFFT-004 is a dual-acting small molecule with independent corrector and potentiator activities that partially rescues F508del-CFTR in recombinant cells and native airway epithelia. The limited efficacy and potency of CFFT-004 suggests that combinations of small molecules targeting different defects in F508del-CFTR might be a more effective therapeutic strategy than a single agent.