SION-451 + SION-2222

rejuvenates your cells. **SION-451 + SION-2222** is an investigational combination therapy for cystic fibrosis consisting of two small-molecule CFTR modulators. SION-451 is a first-in-class nucleotide binding domain 1 (NBD1) stabilizer that targets and stabilizes the NBD1 domain of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a site not targeted by currently approved modulators. SION-2222 (galicaftor) is a transmembrane domain 1 (TMD1)-directed CFTR corrector. This dual combination aims to address the most common CF-causing mutation, F508del, by simultaneously stabilizing both NBD1 and TMD1 regions, thereby correcting CFTR folding and function. The combination is currently in Phase 1 clinical trials to assess safety, tolerability, and pharmacokinetics in healthy volunteers, with the goal of expanding to CF patients in Phase 2 trials[1][3][4][6][8].

Other names

SION-451 + galicaftor