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SION-109
- Development stage
- Unknown
- Lead developer
- Sionna Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
SION-109 is an investigational small molecule corrector developed by Sionna Therapeutics for the treatment of cystic fibrosis. It is designed to target the interface between intracellular loop 4 (ICL4) and nucleotide-binding domain 1 (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. By binding at this site, SION-109 aims to stabilize defective CFTR protein—particularly in patients with the common F508del mutation—helping restore its function as a chloride channel on epithelial cell surfaces. This mechanism is complementary to other NBD1 stabilizers and may enable combination therapies that achieve more complete correction of CFTR dysfunction[1][2][3][5][6][7].
- Brand names
- SION-109SION109SION 109
- Other names
- SION-109SION109SION 109
02
Targets
CFTR (Cystic fibrosis transmembrane conductance regulator)
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