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SION-2847
- Development stage
- Preclinical
- Lead developer
- Sionna Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
SION-2847 is a preclinical small molecule CFTR modulator being developed by Sionna Therapeutics for the treatment of cystic fibrosis. It specifically targets the second transmembrane domain (TMD2) of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. By binding to TMD2, SION-2847 acts as a complementary modulator to enhance the folding, stability, and trafficking of the CFTR protein to the cell surface, particularly in patients with the F508del mutation. It is intended to be used as part of a combination therapy with other modulators, such as NBD1 stabilizers, to achieve full restoration of CFTR function.
02
Targets
CFTR (Cystic fibrosis transmembrane conductance regulator)
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