Filter the drugs by the phase of research, how they help and the responsive mutations.

Cystic fibrosis is a genetic disease caused by mutations in the CFTR gene. Browse the database of drugs treating specific mutations compiled by the AIM tool.
Drug Marketed as Description Phase of research How it helps
VX-440

CFTR corrector

Phase Two Clinical Trial Restore CFTR Function
VX-659

CFTR corrector

Phase Two Clinical Trial Restore CFTR Function
Vancomycin AeroVanc™

Antimicrobial activity

Phase Three Clinical Trial Anti-Infective
Vardenafil

CFTR activator

FDA-/EMA-approved for Other Purpose Restore CFTR Function
Veliparib

PARP-1 inhibitor able to partially restore CFTR channel activity

Pre-clinical Restore CFTR Function
Verotoxin

Rescues endogenous misfolded proteins

Pre-clinical Restore CFTR Function
pGM169/GL67A

Plasmid DNA encoding the CFTR gene

Phase Two Clinical Trial Restore CFTR Function