Corrector combination therapies for F508del-CFTR


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Abstract

HighlightsCombining correctors can increase F508del CFTR functional expression >2.5-fold compared to the best corrector alone.Understanding combination mechanisms of action require multiple research approaches.Combinations may increase drug interactions and reduce need for precision medicine.These are exciting times in the development of therapeutics for cystic fibrosis (CF). New correctors and potentiators of the cystic fibrosis transmembrane conductance regulator (CFTR) are being developed in academic laboratories and pharmaceutical companies, and the field is just beginning to understand their mechanisms of action. Studies of CFTR modulators are also yielding insight into the general principles and strategies that can be used when developing pharmacological chaperones, a new class of drugs. Combining two or even three correctors with a potentiator is an especially promising approach which should lead to further improvements in efficacy and clinical benefit for patients.

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