CFTR modulators<< Back to Clinical management of CF
This section focuses on the use of CFTR modulators for the clinical management of CF. Before exploring more about CFTR modulators it is important to recognise that several disease-modifying strategies are being investigated to treat the underlying causes of CF — CFTR modulators account for just one of these strategies.
The figure below provides an overview of strategies that are currently being investigated to treat the underlying causes of CF.
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- Gene therapy aims to insert non-mutant copies of the CFTR gene into target cells.1
- Non-CFTR ion channel agents are small molecules intended to normalise sodium and chloride transport by targeting non-CFTR ion channels expressed in epithelial cells.2
- Osmotic agents are inhaled hypertonic solutions that restore airway surface liquid by ‘drawing’ liquid out of the airway epithelium into the mucus.3
- CFTR modulators – the focus of this module – are pharmaceutical agents that target specific defects caused by mutations in the CFTR gene. They do not correct errors in the CFTR gene itself (as with gene therapy), but rather, errors that occur from transcription onwards.4
Learn more about the mechanism of action of CFTR modulators.
Explore CFTR modulators that are currently approved for clinical use, including an overview of clinical trial results.