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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.
Praxis Precision Medicines Inc is a clinical-stage biopharmaceutical company. The firm is engaged in translating genetic insights into the development of therapies for patients affected by central nervous system disorders characterized by a neuronal excitation-inhibition imbalance. The company has generated three clinical-stage product candidates, ulixacaltamide (formerly known as PRAX-944), vormatrigine (formerly known as PRAX-628), and relutrigine (formerly known as PRAX-562), as well as PRAX-020 which has been in-licensed by UCB Biopharma SRL, or UCB.