Page Title
Drug Development Pipeline
SION-719
Status
Phase OneTherapeutic Approach
Restore CFTR Protein
SION-719 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 nucleotide binding domain 1, or NBD1. Once the stabilized CFTR protein reaches the cell surface, it forms a channel to allow chloride and sodium (salt) to move in and out of the cell. This new, unique approach could eventually offer an alternative to currently approved modulators.
Status
A Phase 1 study to test the safety and tolerability of SION-718 in healthy volunteers is currently underway in Australia.
Sponsor
This program is sponsored by Sionna Therapeutics and partially funded by the Cystic Fibrosis Foundation.

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