Sionna Therapeutics, Inc. SION

We are a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis ("CF") by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator ("CFTR") protein to deliver clinically meaningful benefit to people with CF. CF is a progressive and life-threatening genetic disease caused by inherited mutations in the CFTR gene, which lead to insufficient CFTR function. While advances in the discovery and development of CFTR modulators have significantly improved the lives of people with CF, at least two-thirds of patients on the current standard of care do not have normal CFTR function, defined as sweat chloride levels below 30 mmol/L. Patients with reduced CFTR function can experience debilitating multi-system complications that lead to significantly impaired quality of life and shorter life expectancy. Our goal is to deliver differentiated medicines for people with CF that can restore their CFTR function to as close to normal as possible by directly stabilizing CFTR's nucleotide binding domain 1 ("NBD1"). Despite having long been identified as a critical component for proper CFTR function, NBD1 has been considered "undruggable," and none of the currently approved CF therapies directly stabilizes NBD1. Leveraging more than a decade of our co-founders' research on NBD1, we are advancing a pipeline of small molecules engineered to correct the defects caused by the F508del genetic mutation, the most common CFTR mutation, which occurs in the NBD1 domain. Approximately 90% of people with CF carry at least one copy of the F508del genetic mutation.