| DC70796 |
SRI-41315
|
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. |
| DC47935 |
NJH-2-057
|
NJH-2-057 is an EN523 OTUB1 recruiter linked to lumacaftor, a drug used to treat cystic fibrosis that binds ΔF508-CFTR. |
| DC12003 |
Corr4A
|
A small molecule corrector of ΔF508-CFTR with IC50 of 6.0 uM.. |
| DC72875 |
GLPG2451
|
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. |
| DC72358 |
(R)-Olacaftor
|
(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). |
| DC72212 |
CFTR corrector 8
|
CFTR corrector 8 is a potent CFTR modulator. CFTR can be used in the research of cystic fibrosis. |
| DC72048 |
SRI-37240
|
SRI-37240 is a potent premature termination codons (PTCs) inhibitor. SRI-37240 suppresses CFTR nonsense mutations. SRI-37240 alters cellular translation termination at PTCs in HEK293T cells. SRI-37240 can also restore CFTR function in primary bronchial epithelial cells when combination with G418. |
| DC70237 |
Bamocaftor potassium
|
Bamocaftor (VX-659) is a next-generation CFTR corrector, restores F508del-CFTR protein function. |
| DC70204 |
ARN23765
|
ARN23765 (ARN 23765) is a highly potent, pharmacological corrector of the mutant CFTR chloride channel with EC50 of 38 pM in cellular assays;
ARN23765 is more than 5000-fold lower compared to presently available corrector drugs.
ARN23765 also showed high efficacy, synergy with other types of correctors, and compatibility with chronic VX-770 potentiator. |
| DC48190 |
Vanzacaftor
|
Vanzacaftor is a modulator of cystic fibrosis transmembrane conductance regulator (CFTR) for treating cystic fibrosis. |
