Novel caveolin modulators as therapeutics for uveitis

发明内容（由申请人提供）：作为葡萄膜炎治疗剂的新型小窝蛋白调节剂项目概述本研究计划寻求开发一种基于经由小窝调节炎症信号传导的葡萄膜炎新疗法。葡萄膜炎导致5-20%的失明，影响工作年龄的人，并在慢性病患者中产生显著的残疾。已知小窝蛋白调节剂如cavtratin能够调节炎症信号传导，包括水肿和白细胞迁移，这是葡萄膜炎中致病级联的关键要素。E&B Technologies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可以预防小鼠和兔葡萄膜炎模型中的视网膜炎症，并在第二阶段完成IND使能工作。从长远来看，将继续进行人体临床试验的概念验证，并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提供一种新的治疗选择， 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