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Sionna Therapeutics Inc is a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) patients by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein to deliver clinically meaningful benefit to CF patients. CF is a progressive and life-threatening genetic disease caused by inherited mutations in the CFTR gene, which heads to insufficient CFTR function. The Company is managed as one operating segment focused on the research and development of cystic fibrosis therapies.
Cogent Biosciences Inc a biotechnology company focused on developing precision therapies for genetically defined diseases. It designs rational precision therapies that treat the underlying cause of disease and improve the lives of patients. Its program CGT9486, is a selective tyrosine kinase inhibitor designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. In the vast majority of cases, KIT D816V is responsible for driving Systemic Mastocytosis (SM), a serious disease caused by the unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with dependence on oncogenic KIT signaling.