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
STAT1

CFTR corrector

Pre-clinical Restore CFTR Function
TCP10L

CFTR corrector

Pre-clinical Restore CFTR Function
TFG

CFTR corrector

Pre-clinical Restore CFTR Function
TMA

CFTR corrector and potentiator

Pre-clinical Restore CFTR Function
Thymosin α1 ZADAXIN™

Natural polypeptide reducing inflammation and increasing CFTR maturation, stability and activity

Therapeutic not approved by FDA or EMA Restore CFTR Function + Anti-Inflammatory
VX-152

CFTR corrector

Phase Two Clinical Trial Restore CFTR Function
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
Veliparib

PARP-1 inhibitor able to partially restore CFTR channel activity

Pre-clinical Restore CFTR Function