10.3389/fphar.2019.00514.s001
Gert de Wilde
Gert
de Wilde
Maarten Gees
Maarten
Gees
Sara Musch
Sara
Musch
Katleen Verdonck
Katleen
Verdonck
Mia Jans
Mia
Jans
Anne-Sophie Wesse
Anne-Sophie
Wesse
Ashvani K. Singh
Ashvani K.
Singh
Tzyh-Chang Hwang
Tzyh-Chang
Hwang
Thierry Christophe
Thierry
Christophe
Mathieu Pizzonero
Mathieu
Pizzonero
Steven Van der Plas
Steven
Van der Plas
Nicolas Desroy
Nicolas
Desroy
Marlon Cowart
Marlon
Cowart
Pieter Stouten
Pieter
Stouten
Luc Nelles
Luc
Nelles
Katja Conrath
Katja
Conrath
Data_Sheet_1_Identification of GLPG/ABBV-2737, a Novel Class of Corrector, Which Exerts Functional Synergy With Other CFTR Modulators.pdf
Frontiers
2019
cystic fibrosis transmembrane conductance regulator (CFTR)
cystic fibrosis
chloride channel
electrophysiology
protein misfolding
2019-05-09 12:20:23
Dataset
https://frontiersin.figshare.com/articles/dataset/Data_Sheet_1_Identification_of_GLPG_ABBV-2737_a_Novel_Class_of_Corrector_Which_Exerts_Functional_Synergy_With_Other_CFTR_Modulators_pdf/8100470
<p>The deletion of phenylalanine at position 508 (F508del) in cystic fibrosis transmembrane conductance regulator (CFTR) causes a severe defect in folding and trafficking of the chloride channel resulting in its absence at the plasma membrane of epithelial cells leading to cystic fibrosis. Progress in the understanding of the disease increased over the past decades and led to the awareness that combinations of mechanistically different CFTR modulators are required to obtain meaningful clinical benefit. Today, there remains an unmet need for identification and development of more effective CFTR modulator combinations to improve existing therapies for patients carrying the F508del mutation. Here, we describe the identification of a novel F508del corrector using functional assays. We provide experimental evidence that the clinical candidate GLPG/ABBV-2737 represents a novel class of corrector exerting activity both on its own and in combination with VX809 or GLPG/ABBV-2222.</p>