Novel caveolin modulators as therapeutics for uveitis

描述（由申请人提供）：新型小泡蛋白调节剂作为葡萄膜炎的治疗药物项目概述本研究计划旨在开发一种基于通过小泡调节炎症信号的新型葡萄膜炎治疗方法。葡萄膜炎导致5-20%的失明，影响到工作年龄的人，并对慢性疾病患者造成严重残疾。众所周知，cavtratin等腔洞蛋白调节剂能够调节炎症信号，包括水肿和白细胞迁移，这是葡萄膜炎致病级联反应的关键因素。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的视网膜炎症，并在II期完成IND支持工作。从长远来看，将在人体临床试验中进行概念验证，并进行监管部门批准所需的临床试验。这个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，减少失明

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