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GLPG2451 + galicaftor (GLPG2222) is an investigational dual combination therapy developed by Galapagos NV, in collaboration with AbbVie, for the treatment of cystic fibrosis. The combination consists of GLPG2451, a novel CFTR potentiator, and galicaftor (GLPG2222), a CFTR corrector. These small molecule agents are designed to work synergistically to address the underlying protein defect in cystic fibrosis by improving both the trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) protein to the cell surface and its gating activity once there. The combination has been evaluated in Phase 1 studies in healthy volunteers and in the Phase 1b FALCON trial involving adult patients homozygous for the F508del mutation. Clinical data indicated that the dual therapy could reduce sweat chloride concentrations and improve lung function as measured by ppFEV1.
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