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 是一家初创制药公司，专注于开发新型“纳米海绵”链接 用于治疗胆管癌 (CCA) 的小分子 DCLK1 抑制剂，这是一种罕见的侵袭性癌症 晚期胆管恶性肿瘤，预后不佳。 CCA的发生率 随着年龄的增长，CCA 的 5 年生存率为 13%（中位生存期为 7 个月）。发病率 CCA 正在增加，DCLK1 LLC 估计 2022 年美国发病率将超过 10,865 例。 化疗是目前 CCA 患者围手术期治疗的标准治疗方法。 DCLK1 LLC 正在与 KUMC 研究人员合作开发一种新的 CCA 治疗策略。 双皮质素 钙调蛋白样激酶 1 (DCLK1) 是一种丝氨酸-苏氨酸激酶，代表一种新型治疗方法 肿瘤学目标。我们的团队已经证明 DCLK1 表达的敲低和小分子 DCLK1 抑制剂可抑制小鼠异种移植肿瘤的生长。 DCLK1 LLC 最大限度地提高 DCLK1 抑制剂的功效和安全性的策略是提供这些 使用合理设计的肽将有效分子定位到胆道肿瘤部位 纳米海绵技术。这两种独特的技术结合在一起提供了可能性 直接对肝脏进行突破性治疗并改变治疗模式 CCA 患者。