SBIR CONCEPT AWARD-NANOSPONGE LINKED DCLK1 INHIBITORS FOR THE TARGETED TREATMENT OF CHOLANGIOCARCINOMA

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DCLK1 LLC is a startup pharmaceutical company focused on developing novel "nano-sponge" linked small molecule DCLK1 inhibitors for treating cholangiocarcinoma (CCA), a rare and aggressive malignancy of the bile ducts that presents at a late stage with poor outcomes. The incidence of CCA increases with age with a 5-Year survival of CCA is 13% (median survival 7 months). Incidence of CCA is increasing and DCLK1 LLC estimates more than 10,865 US incidence in 2022. Cytotoxic chemotherapy is currently the standard of care for CCA patients treated perioperatively. DCLK1 LLC is collaborating with KUMC researchers on a novel treatment strategy for CCA. Doublecortin Calmodulin-like Kinase 1 (DCLK1) is a serine-threonine kinase which represents a novel therapeutic target in oncology. Our team has shown that both knockdown of DCLK1 expression and small molecule DCLK1 inhibitors suppress xenograft tumor growth in mice. DCLK1 LLC's strategy to maximize the efficacy and safety of DCLK1 inhibitors is to deliver these potent molecules to the site of the tumors in the biliary tract using a rationally designed peptide nanosponge technology. Taken together these two unique technologies offer the possibility of administering breakthrough treatment directly to the liver and changing the treatment paradigm for CCA patients.