SION-109 is a corrector, a type of CFTR modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. It aims to stabilize defective CFTR by targeting a specific location on the protein called intracellular loop 4, or ICL4. Once the stabilized CFTR protein reaches the cell surface, it forms a channel to allow chloride and sodium (salt) to move in an out of the cell. This approach could eventually offer an alternative to currently approved modulators to correct this aspect of the CFTR protein.

Status

A Phase 1 study to test the safety and tolerability of SION-109 in healthy volunteers is currently underway. Future studies are planned to evaluate SION-109 in people with CF who have the F508del CFTR mutation.

Sponsor

This program is sponsored by Sionna Therapeutics and partially funded by the Cystic Fibrosis Foundation.