sionna therapeutics

Sionna Therapeutics is a clinical-stage biopharmaceutical company on a mission to revolutionize the treatment paradigm for cystic fibrosis (CF) patients. The company focuses on developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein to deliver clinically meaningful benefits to CF patients. Their goal is to deliver differentiated medicines that can restore CFTR function as close to normal as possible by directly stabilizing CFTR's nucleotide-binding domain 1 (NBD1). Sionna is currently conducting Phase 1 trials for two highly potent NBD1 stabilizers and developing a portfolio of complementary CFTR modulators designed to work synergistically with their NBD1 stabilizers to improve CFTR function, as observed in preclinical models.