1. Abela AR, Alcacio T, Anderson C, Angell PT, Baek M, Clemens JJ, Cleveland T, Ferris LA, Grootenhuis PDJ, Gross RS et al.. (2018) Modulator of cystic fibrosis transmembrane conductance regulator, pharmaceutical compositions, methods of treatment, and process for making the modulator.
Patent number: WO2018107100A1. Assignee: Vertex Pharmaceuticals. Priority date: 09/12/2016. Publication date: 14/04/2019.
2. Alcacio T, Baek M, Grootenhuis P, Hadida Ruah SS, Hughes RM, Keshavarz-Shokri A, McAuley-Aoki R, McCartney J, Miller MT, Van Goor F et al.. (2018) Modulator of cystic fibrosis transmembrane conductance regulator, pharmaceutical compositions, methods of treatment, and process for making the modulator.
Patent number: WO2018064632A1. Assignee: Vertex Pharmaceuticals. Priority date: 30/09/2016. Publication date: 05/04/2018.
3. Davies JC, Moskowitz SM, Brown C, Horsley A, Mall MA, McKone EF, Plant BJ, Prais D, Ramsey BW, Taylor-Cousar JL et al.. (2018) VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles.
N Engl J Med, 379 (17): 1599-1611.
[PMID:30334693]
4. Grand DL, Gosling M, Baettig U, Bahra P, Bala K, Brocklehurst C, Budd E, Butler R, Cheung AK, Choudhury H et al.. (2021) Discovery of Icenticaftor (QBW251), a Cystic Fibrosis Transmembrane Conductance Regulator Potentiator with Clinical Efficacy in Cystic Fibrosis and Chronic Obstructive Pulmonary Disease.
J Med Chem, [Epub ahead of print].
[PMID:34028270]
5. Keating D, Marigowda G, Burr L, Daines C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Sass LA, Tullis E et al.. (2018) VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles.
N Engl J Med, 379 (17): 1612-1620.
[PMID:30334692]
6. Pedemonte N, Boido D, Moran O, Giampieri M, Mazzei M, Ravazzolo R, Galietta LJ. (2007) Structure-activity relationship of 1,4-dihydropyridines as potentiators of the cystic fibrosis transmembrane conductance regulator chloride channel.
Mol Pharmacol, 72 (1): 197-207.
[PMID:17452495]
7. Pesci E, Bettinetti L, Fanti P, Galietta LJ, La Rosa S, Magnoni L, Pedemonte N, Sardone GL, Maccari L. (2015) Novel Hits in the Correction of ΔF508-Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Protein: Synthesis, Pharmacological, and ADME Evaluation of Tetrahydropyrido[4,3-d]pyrimidines for the Potential Treatment of Cystic Fibrosis.
J Med Chem, 58 (24): 9697-711.
[PMID:26561003]
8. Ruah SSH, Grootenhuis PDJ, Van Goor F, Zhou J, Bear B, Miller MT, McCartney J, Numa MMD. (2009) Indole derivatives as CFTR modulators.
Patent number: US20090131492A1. Assignee: ertex Pharmaceuticals Inc. Priority date: 07/04/2006. Publication date: 21/05/2009.
9. Scanio MJC, Searle XB, Liu B, Koenig JR, Altenbach R, Gfesser GA, Bogdan A, Greszler S, Zhao G, Singh A et al.. (2019) Discovery of ABBV/GLPG-3221, a Potent Corrector of CFTR for the Treatment of Cystic Fibrosis.
ACS Med Chem Lett, 10 (11): 1543-1548.
[PMID:31749908]
10. Sheppard DN, Welsh MJ. (1992) Effect of ATP-sensitive K+ channel regulators on cystic fibrosis transmembrane conductance regulator chloride currents.
J Gen Physiol, 100 (4): 573-91.
[PMID:1281220]
11. Tradtrantip L, Namkung W, Verkman AS. (2010) Crofelemer, an antisecretory antidiarrheal proanthocyanidin oligomer extracted from Croton lechleri, targets two distinct intestinal chloride channels.
Mol Pharmacol, 77 (1): 69-78.
[PMID:19808995]
12. Verkman AS, Galietta LJ. (2009) Chloride channels as drug targets.
Nat Rev Drug Discov, 8 (2): 153-71.
[PMID:19153558]