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Functional and biochemical characterization of Alyftrek components reveals high-potency rescue of F508del-CFTR

Year: 2026
Type: article
Abstract: Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) was evaluated in cystic fibrosis (CF) airway models. S-vanzacaftor showed high potency, restoring F508del-CFTR function at nanomolar concentrations, enhanced by tezacaftor. Deutivacaftor matched ivacaftor efficacy in triple combinations. Both regimens improved CFTR maturation. The R-enantiomer was less effective and antagonistic. Notably, reduced doses retained substantial activity, supporting lower dosing strategies. These findings highlight S-vanzacaftor potency and validate deutivacaftor as an effective potentiator in CF therapy.
DOI: 10.1152/ajplung.00103.2026
Authors: Sandra Mirval, Louise Damy, Auguste Sauvage-de-Gérando, Christine Barrault, Isabelle Fixe, Edouard Sage, Christelle Coraux, Benjamin Simonneau, Bénédicte Duriez, Pascale Fanen, Anne Cantereau, Clarisse Vandebrouck, Frédéric Becq
Institutions: Université de Poitiers, Université de Versailles Saint-Quentin-en-Yvelines, Hôpital Foch, Université de Reims Champagne-Ardenne, Inserm, Université Paris-Est Créteil
Language: English
Field: Medicine
Open Access status: hybrid
OpenAlex ID: W7203741362
FWCI:
Cites: 40
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Related to: 10