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SION-2849

Development stage: Preclinical
Lead developer: Sionna Therapeutics
Modality: Small Molecules

Overview

SION-2849 is a small molecule CFTR modulator being developed by Sionna Therapeutics for the treatment of cystic fibrosis. It specifically targets the first transmembrane domain (TMD1) of the CFTR protein, acting as a corrector to improve the folding, stability, and trafficking of the protein to the cell surface. SION-2849 is designed to be used as a complementary modulator in combination with other CFTR-targeting agents, such as NBD1 stabilizers, to enhance the functional correction of the CFTR channel in patients with the F508del mutation. The drug is currently in the preclinical stage of development.

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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