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
Gene editing via CRISPR Cas9/gRNA

Corrects aberrant splicing signals

Pre-clinical Restore CFTR Function
Hepatocyte growth factor

Enhances functional rescue of Phe508del-CFTR

Pre-clinical Restore CFTR Function
HspA4

CFTR corrector

Pre-clinical Restore CFTR Function
Hypertonic Saline

Improves mucus clearance by increasing hydration

Therapeutic not approved by FDA or EMA Mucociliary Clearance
IGF-1

Enhances protein expression of CFTR

Pre-clinical Restore CFTR Function
IPDMA 

CFTR corrector and potentiator

Pre-clinical Restore CFTR Function
IPEMA

CFTR corrector and potentiator

Pre-clinical Restore CFTR Function
KB001

Targets a Pseudomonas aeruginosa virulence factor

Phase Two Clinical Trial Anti-Inflammatory
MCG1516A

CFTR corrector

Pre-clinical Restore CFTR Function
MG-132

Proteasome inhibitor preventing premature CFTR breakdown

Pre-clinical Restore CFTR Function