C18

Small molecule CFTR corrector

Phase of research

Pre-clinical

How it helps

Restore CFTR Function

General information

C18 is a small-molecule CFTR corrector discovered by Vertex Pharmaceuticals. It is an experimental substance used in studies and not yet available as a human pharmaceutical. In relation to cystic fibrosis, our AIM tool found the data that C18 can correct various CFTR mutations. 

Synonyms

N/A

Marketed as

N/A

Dietary sources

N/A

Structure image not available

N/A

Drug-Mutation Relation

Treats

Mutation Number of sources Average impact factor
A455E
1 2.85
Link Tested on Impact factor Notes
Correctors Rescue CFTR Mutations in Nucleotide-Binding Domain 1 (NBD1) by Modulating Proteostasis human cell cultures 2.85 C18 on its own is more effective than in combination with C3. Better in combination with C4.
F508del
2 2.81
Link Tested on Impact factor Notes
Increasing the Endoplasmic Reticulum Pool of the F508del Allele of the Cystic Fibrosis Transmembrane Conductance Regulator Leads to Greater Folding Correction by Small Molecule Therapeutics human cell cultures 2.81 Better in combination with PYR-41.
Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis human cell cultures N/A
R560T
1 2.85
Link Tested on Impact factor Notes
Correctors Rescue CFTR Mutations in Nucleotide-Binding Domain 1 (NBD1) by Modulating Proteostasis human cell cultures 2.85 Little effect on its own or in combination with C3. Better in combination with C4.
S492F
1 2.85
Link Tested on Impact factor Notes
Correctors Rescue CFTR Mutations in Nucleotide-Binding Domain 1 (NBD1) by Modulating Proteostasis human cell cultures 2.85 Better in combination with C4.
ΔI507
1 2.85
Link Tested on Impact factor Notes
Correctors Rescue CFTR Mutations in Nucleotide-Binding Domain 1 (NBD1) by Modulating Proteostasis human cell cultures 2.85 Little effect on its own or in combination with C3. Better in combination with C4.

Does not treat

Mutation Number of sources Average impact factor
A561E
1 0.00
Link Tested on Impact factor Notes
Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis human cell cultures N/A