GLPG2451 (GLPG 2451) is a potent potentiator of cystic fibrosis transmembrane conductance regulator (CFTR) with EC50 of 11 nM for F508del CFTR in the YHA assay and EC50 of 18 nM in TECC assay using HBE cells from patients carrying the F508del mutation.
GLPG2451 improves the gating function of the CFTR channel.
SRI-41315 is a small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion, restores CFTR expression and function, suppresses CFTR nonsense mutations.SRI-41315 induces translational readthrough by depleting eRF1 protein level and prolonging the translational pause that occurs at premature termination codons (PTCs).SRI-41315 reduced eRF1 levels in a manner dependent upon a ubiquitin-mediated proteasome degradation pathway.SRI-41315 in combination with G418 restores CFTR function in primary bronchial epithelial cells derived from a CF patient with CFTR nonsense alleles.
Galicaftor (ABBV-2222/GLPG-2222) is an orally bioavailable small molecule that functions as a CFTR corrector, demonstrating potential for therapeutic development in cystic fibrosis research.
Crinecerfont tosylate (SSR-125543 tosylate) hydrochloride is a potent, orally active, non-peptide CRF1 receptor antagonist. Crinecerfont tosylate can be used for Classic congenital adrenal hyperplasia (CAH) research. Crinecerfont tosylate is a click chemistry reagent, it contains an Alkyne group and can undergo copper-catalyzed azide-alkyne cycloaddition (CuAAc) with molecules containing Azide groups.
CFTR corrector 18 (Compound I-99) is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector. CFTR corrector 18 facilitates the processing and trafficking of CFTR, increasing the number of CFTR on the cell surface. CFTR corrector 18 is promising for research of cystic fibrosis (CF).
(R)-Olacaftor ((R)-VX-440) is a Cystic fibrosis transmembrane conductance regulator (CFTR) modulator. (R)-Olacaftor has good potential for the study of cystic fibrosis (CF).