SION-451
Cystic Fibrosis (F508del mutation)
Key Facts
About sionna-therapeutics
Sionna Therapeutics is on a mission to revolutionize cystic fibrosis treatment by developing novel small molecules that directly stabilize the NBD1 domain of the CFTR protein, the site of the most common disease-causing mutation. Leveraging over a decade of foundational research, the company has advanced a pipeline of NBD1 stabilizers and complementary modulators into clinical development, aiming to normalize CFTR function where current standards of care fall short. Its strategy is to build a dominant CF franchise by developing these candidates as potential best-in-class therapies, either alone or in combination, to address the significant unmet need for patients who remain underserved by existing modulator regimens.
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