SION-453

SION-453 is a small molecule CFTR modulator being developed by Sionna Therapeutics for the treatment of cystic fibrosis. It is specifically designed to stabilize the first nucleotide-binding domain (NBD1) and its interaction with the fourth intracellular loop (ICL4) of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. This dual-targeting approach aims to correct the misfolding and degradation of the CFTR protein, particularly in patients with the common F508del mutation, thereby increasing the amount of functional protein that reaches the cell surface. SION-453 is currently being evaluated in Phase 1 clinical trials as a complementary modulator intended for use in combination regimens to enhance therapeutic efficacy over existing standards of care.