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galicaftor

Development stage: Phase 2
Lead developer: Sionna Therapeutics
Modality: Small Molecules
Administration: Oral

Overview

Galicaftor is a potent, orally active small molecule that acts as a cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It is designed to improve the folding and trafficking of defective CFTR protein to the cell surface, thereby enhancing chloride ion transport in patients with cystic fibrosis. Galicaftor has been investigated primarily for the treatment of cystic fibrosis, often in combination with other CFTR modulators. The drug was originally developed by Galapagos NV and AbbVie, with recent licensing activity involving Sionna Therapeutics. Its mechanism targets correction of misfolded CFTR protein rather than potentiation of channel gating[1][3][4][5].

Other names: galicaftor [INN]1918143-53-9

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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