Preventing choroidal neovascularization by long-term transscleral drug delivery

描述(申请人提供)：年龄相关性黄斑变性(ARMD)是生活在工业化世界的55岁以上人群的主要致盲原因。随着新型抗血管生成药物的引入，目前临床干预是可能的，但仅限于湿性疾病。然而，这些治疗方法受到严重限制，因为它们必须直接注射到眼睛里。这种手术有很大的并发症风险，并产生可以理解的患者可接受性问题。显然需要一种治疗方法，要么绕过眼部注射的需要，要么减少注射的频率。经巩膜外给药为眼球后段给药提供了一种方便的途径。我们建议测试和开发一种新颖而强大的跨巩膜给药系统，该系统有望实现无毒、微创和高度可定制的不同治疗方法。这项提议的成功将为制药业和世界各地的患者提供一种新的方法来治疗ARMD，而不需要目前每月或6周眼内注射的负担。对于概念验证，我们将使用一种新颖的、基于人类蛋白质的疗法，该疗法已在初步动物研究中显示出巨大的潜力。

DESCRIPTION (provided by applicant): Age-related Macular Degeneration (ARMD) is the leading cause of blindness for individuals over fifty-five years of age that live in the industrialized world. With the introduction of novel anti-angiogenic agents, clinical intervention is currently possible, but only for the wet form of the disease. However, these treatments are severely limited by the fact that they must be directly injected into the eye. This procedure carries a significant risk of complication and generates understandable issues of patient acceptability. There is a clear need for a therapy that either bypasses the need for ocular injection or reduces its frequency. Transscleral drug delivery provides a convenient way for delivering drug to the posterior segment of the eye. We propose to test and develop a novel and powerful transscleral drug delivery system that holds promise for being non-toxic, minimally invasive and highly customizable towards different therapies. The success of this proposal will offer a new method to the pharmaceutical industry and to patients worldwide to treat ARMD without the current burden of monthly or 6-weekly intraocular injections. For proof-of-concept, we will use a novel, human protein-based therapy that has shown great potential in preliminary animal studies.