Target intelligence / Profile preview

Cystic Fibrosis Transmembrane Conductance Regulator (None)

Target: None
Molecular classification: ABC transporter-class ion channel, Transporter

Overview

The cystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and anion channel that functions primarily as a chloride and bicarbonate ion channel across epithelial cell membranes. Dysfunction of CFTR leads to cystic fibrosis, a common lethal genetic disease. Recent advances include CFTR modulators, small molecule drugs that either potentiate defective channels or increase their presence at the cell surface, improving clinical outcomes.

Mechanism of action

CFTR modulators potentiate defective channels or increase their presence at the cell surface.

Biological functions

Chloride ion transportBicarbonate secretionWater movement regulationRegulation of sodium channels

Disease associations

Cystic fibrosisCOPDAsthmaBronchiectasisAllergic bronchopulmonary aspergillosisMale infertility (due to congenital absence of vas deferens)

Safety considerations

Specific safety concerns vary depending on the modulator usedPotential for acquired dysfunction due to environmental factors

Interacting drugs

Ivacaftor

3 more in the full profile.

Biomarkers

Sweat chloride levels

Beyond the preview

Go deeper on Cystic Fibrosis Transmembrane Conductance Regulator (None).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Explore the programs pursuing this target and their development progress.

Drug candidates
Developers
Development stage

Clinical trials

Follow the clinical studies evaluating therapies directed at this target.

Trial design
Status
Readouts

Competitive landscape

Compare approaches across drug candidates, modalities, and indications.

Programs
Modalities
Indications

Literature & evidence

Investigate the research and source evidence behind target biology and development.

Publications
Sources
Analysis

Patents

Explore patent activity around therapies and technologies addressing this target.

Patents
Assignees
Technologies

Research & analysis

Connect target biology, drug development, and emerging evidence in your research.

Biology
Development news
Analysis

Bring the full picture into focus.

See how Gosset can support your research on Cystic Fibrosis Transmembrane Conductance Regulator (None).

Explore the full profile