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

SBIR 概念奖-纳米海绵连接的 DCLK1 抑制剂用于胆管癌的靶向治疗

• 批准号: 10975720
• 负责人: THAIS SIELECKI
• 金额: $ 35.34万
• 依托单位: DCLK1 LLC
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-02-28 至 2024-02-27
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10975720
• 关键词: SBIR; CONCEPT; AWARD; NANOSPONGE; LINKED

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.

DCLK1 LLC是一家企业制药公司 用于治疗胆管癌（CCA）的小分子DCLK1抑制剂，这是一种罕见而侵略性的 胆管的恶性肿瘤在后期呈现出较差的结果。 CCA的发生率 随着年龄的增长，CCA的5年生存率为13％（中位生存期7个月）。发病率 CCA正在增加，DCLK1 LLC估计2022年美国发病率超过10,865。 化学疗法目前是围手术期治疗的CCA患者的护理标准。 DCLK1 LLC正在与KUMC研究人员合作，以解决CCA的新治疗策略。 Doublecortin 钙调蛋白样激酶1（DCLK1）是一种丝氨酸 - 硫代激酶，代表一种新型治疗 肿瘤学目标。我们的团队表明，DCLK1表达和小分子的敲低 DCLK1抑制剂抑制小鼠异种移植肿瘤的生长。 DCLK1 LLC最大化DCLK1抑制剂的功效和安全性的策略是提供这些 使用合理设计的肽，有效分子到胆道中的肿瘤部位 纳米传播技术。总结这两种独特的技术提供了可能性 直接对肝脏进行突破性治疗，并更改治疗范例 CCA患者。