Novel caveolin modulators as therapeutics for uveitis

描述(由申请人提供)：作为葡萄膜炎治疗药物的新型小窝调节剂本研究计划寻求开发一种基于小窝调节炎症信号的治疗葡萄膜炎的新疗法。葡萄膜炎导致5%-20%的失明，影响到工作年龄的人，并在慢性病患者中造成严重残疾。已知的小窝蛋白调节剂，如Cavtratin，能够调节炎症信号，包括水肿和白细胞迁移，这是葡萄膜炎致病级联的关键因素。E&B科技公司正在开发一种改进的第二代Cavtratin，CVX-001，用于治疗葡萄膜炎。该项目的目标是证明CVX-001可以在小鼠和兔葡萄膜炎模型中预防视网膜炎症，并在第二阶段完成IND使能工作。长期而言，将继续进行人体临床测试的概念验证，并将进行监管批准所需的临床试验。该项目的成功完成将为葡萄膜炎患者建立一种新的治疗选择，减少失明 和视力障碍。1

DESCRIPTION (provided by applicant): Novel caveolin modulators as therapeutics for uveitis Project Summary This research proposal seeks to develop a novel therapy for uveitis based upon modulation of inflammatory signaling via caveolae. Uveitis drives 5-20% of blindness, affecting people of working age and producing significant disability in patients with chronic disease. Caveolin modulators such as cavtratin are known to be able to modulate inflammatory signaling including edema and leukocyte migration, key elements of the pathogenic cascade in uveitis. E&B Technologies is developing an improved, second generation cavtratin, CVX-001, for the treatment of uveitis. The project goal is to demonstrate that CVX-001 can prevent retinal inflammation in mouse and rabbit models of uveitis and, in phase II, IND enabling work completed. Longer term, proof of concept in human clinical testing will be pursued and the clinical trials needed for regulatory approval will be conducted. Successful completion of this project will establish a new therapeutic option for uveitis sufferers with a reduction in blindness and visual impairment. 1