In Vitro Responsiveness of 417 CFTR Variants to Vanzacaftor/Tezacaftor/Ivacaftor. (PubMed, J Cyst Fibros) - Sep 12, 2026 - "We evaluated 417 CFTR variants for VTI responsiveness, including 272 tested for the first time and 145 previously categorized as non-responsive (NR) or borderline-responsive (BR) to ETI. Overall, ∼76% of variants achieved ≥10% of WT improvement, the threshold for responsiveness. This included 87% of newly tested variants and 55% of ETI NR/BR variants. Notably, 50 variants classified as NR/BR to ETI reached the responsive threshold with VTI using empirical Bayes criteria. Compared with ETI, VTI demonstrated broader efficacy across this dataset. These results expand the number of CFTR variants likely to benefit from highly effective modulator therapy and highlight remaining variants that may require alternative therapeutic strategies."
Journal • Preclinical • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases
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Alexi Panagiotou; Andrey Sivachenko; Ashley Baldwin; Divesh Patel; Garry R Cutting; Hermann Bihler; Jan Harrington; Joshua Conte; Karen S Raraigh; Kevin Coote; Kylie Arnold; Martin Mense; Michael J Mulligan; Normand E Allaire; Priyanka Bhatt; Rachel Valdez Misiolek; Violaine Bailey; Yi Cheng
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