Sionna Therapeutics is focused on developing innovative treatments for cystic fibrosis (CF) by targeting the cystic fibrosis transmembrane conductance regulator (CFTR) protein, specifically addressing the defects caused by the F508del mutation, the most prevalent CF mutation. The company is advancing a pipeline of small molecules designed to stabilize the nucleotide-binding domain 1 (NBD1) of CFTR, which is crucial for the protein's proper function and trafficking to the cell surface. By correcting the instability of NBD1, Sionna aims to restore normal CFTR function, which is essential for regulating chloride ion and water flow across epithelial membranes. This approach is expected to provide more effective treatment options compared to existing therapies that only partially improve CFTR function.

In addition to its NBD1 stabilizers, Sionna is also developing complementary modulators that target other critical domains of the CFTR protein, such as the intracellular loop 4 (ICL4) and transmembrane domain 1 (TMD1). The company has entered into a license agreement with AbbVie to incorporate several clinical-stage compounds into its pipeline, enhancing its ability to create dual combination therapies that could potentially offer superior efficacy over current standard treatments. Sionna's strategy is to combine its novel NBD1 stabilizers with these complementary modulators to achieve a more comprehensive correction of CFTR function, ultimately aiming to normalize health for individuals affected by cystic fibrosis.

United States

Sionna Therapeutics is a clinical stage company developing novel small molecule therapies targeting the most common genetic mutation affecting cystic fibrosis (CF) patients. The company is dedicated to developing highly effective and differentiated treatments for cystic fibrosis.

Sionna Therapeutics

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