Modulators of CFTR. Updates on clinical development and future directions

Emmanuelle E Bardin; Alexandra  Pastor; Michaela Semeraro; Anita Golec; Kate Hayes; Benoit Chevalier; Farouk Berhal; Guillaume Prestat; Alexandre Hintzpeter; Christine Gravier-Pelletier; Iwona  Pranke; Isabelle Sermet-Gaudelus

doi:10.1016/j.ejmech.2021.113195

Modulators of CFTR. Updates on clinical development and future directions

Emmanuelle E Bardin, Alexandra Pastor, Michaela Semeraro, Anita Golec, Kate Hayes, Benoit Chevalier, Farouk Berhal, Guillaume Prestat, Alexandre Hintzpeter, Christine Gravier-Pelletier, Iwona Pranke, Isabelle Sermet-Gaudelus^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

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Abstract

Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in the Caucasian population. It is due to mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Current symptomatic CF therapies, which treat the downstream consequences of of CFTR mutations, have increased survival. Better knowledge of the of the CFTR protein has enabled pharmacologic therapy aiming to restore mutated CFTR expression and function. These CFTR "modulators" have revolutionised the CF therapeutic landscape, with the potential to transform prognosis for a considerable number of patients. This review provides a brief summary of their mechanism of action and presents a thorough review of the results obtained from clinical trials of CFTR modulators.

Original language	English
Article number	113195
Number of pages	15
Journal	European Journal of Medicinal Chemistry
Volume	213
Early online date	16 Jan 2021
DOIs	https://doi.org/10.1016/j.ejmech.2021.113195
Publication status	Published - Mar 2021
Externally published	Yes

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Modulators of CFTR. Updates on clinical development and future directions
Copyright 2021 Elsevier. This manuscript is distributed under a Creative Commons Attribution-NonCommercial-NoDerivs License (https://creativecommons.org/licenses/by-nc-nd/4.0/), which permits distribution and reproduction for non-commercial purposes, provided the author and source are cited
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title = "Modulators of CFTR. Updates on clinical development and future directions",

abstract = "Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in the Caucasian population. It is due to mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Current symptomatic CF therapies, which treat the downstream consequences of of CFTR mutations, have increased survival. Better knowledge of the of the CFTR protein has enabled pharmacologic therapy aiming to restore mutated CFTR expression and function. These CFTR {"}modulators{"} have revolutionised the CF therapeutic landscape, with the potential to transform prognosis for a considerable number of patients. This review provides a brief summary of their mechanism of action and presents a thorough review of the results obtained from clinical trials of CFTR modulators. ",

author = "Bardin, {Emmanuelle E} and Alexandra Pastor and Michaela Semeraro and Anita Golec and Kate Hayes and Benoit Chevalier and Farouk Berhal and Guillaume Prestat and Alexandre Hintzpeter and Christine Gravier-Pelletier and Iwona Pranke and Isabelle Sermet-Gaudelus",

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doi = "10.1016/j.ejmech.2021.113195",

language = "English",

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journal = "European Journal of Medicinal Chemistry",

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TY - JOUR

T1 - Modulators of CFTR. Updates on clinical development and future directions

AU - Bardin, Emmanuelle E

AU - Pastor, Alexandra

AU - Semeraro, Michaela

AU - Golec, Anita

AU - Hayes, Kate

AU - Chevalier, Benoit

AU - Berhal, Farouk

AU - Prestat, Guillaume

AU - Hintzpeter, Alexandre

AU - Gravier-Pelletier, Christine

AU - Pranke, Iwona

AU - Sermet-Gaudelus, Isabelle

PY - 2021/3

Y1 - 2021/3

N2 - Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in the Caucasian population. It is due to mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Current symptomatic CF therapies, which treat the downstream consequences of of CFTR mutations, have increased survival. Better knowledge of the of the CFTR protein has enabled pharmacologic therapy aiming to restore mutated CFTR expression and function. These CFTR "modulators" have revolutionised the CF therapeutic landscape, with the potential to transform prognosis for a considerable number of patients. This review provides a brief summary of their mechanism of action and presents a thorough review of the results obtained from clinical trials of CFTR modulators.

AB - Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in the Caucasian population. It is due to mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Current symptomatic CF therapies, which treat the downstream consequences of of CFTR mutations, have increased survival. Better knowledge of the of the CFTR protein has enabled pharmacologic therapy aiming to restore mutated CFTR expression and function. These CFTR "modulators" have revolutionised the CF therapeutic landscape, with the potential to transform prognosis for a considerable number of patients. This review provides a brief summary of their mechanism of action and presents a thorough review of the results obtained from clinical trials of CFTR modulators.

U2 - 10.1016/j.ejmech.2021.113195

DO - 10.1016/j.ejmech.2021.113195

M3 - Article

SN - 0223-5234

VL - 213

JO - European Journal of Medicinal Chemistry

JF - European Journal of Medicinal Chemistry

M1 - 113195

ER -

Modulators of CFTR. Updates on clinical development and future directions

Abstract

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