Vanzacaftor-Tezacaftor as an alternative therapeutic resource for the ETI-Resistant L467F-F508del Allele: Ex vivo prediction and exploratory clinical assessment.

doi:10.1016/j.jcf.2026.05.015

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Article

Vanzacaftor-Tezacaftor as an alternative therapeutic resource for the ETI-Resistant L467F-F508del Allele: Ex vivo prediction and exploratory clinical assessment.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Sept 2026

Capurro Valeria, Pesce Emanuela, Cresta Federico, Pastorino Cristina, Mantero Alice, Tomati Valeria, Daccò Valeria, Claut Laura, Fabrizzi Benedetta, Zara Federico, Castellani Carlo, Pedemonte Nicoletta

Abstract excerpt

BACKGROUND: The triple combination of Elexacaftor/Tezacaftor/Ivacaftor (ETI) has revolutionized cystic fibrosis (CF) treatment; however, a subset of patients with eligible genotypes remains unresponsive. We previously identified the L467F-F508del complex allele as a cause of therapeutic failure, conferring a severe processing defect that renders the CFTR protein refractory to ETI. METHODS: To address this unmet...

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Topics

Humans
Cystic Fibrosis
Quinolones
Cystic Fibrosis Transmembrane Conductance Regulator

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Aminophenols

Benzodioxoles

Indoles

Alleles

Drug Combinations

Female

Chloride Channel Agonists

Drug Resistance

Male

Spiro Compounds

Source

Open in PubMed

Identifiers

PMID: 42243036
DOI: 10.1016/j.jcf.2026.05.015

Genes

CFTR

Provenance

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