A new class of wet AMD therapeutics

摘要 晚期新生血管性老年性黄斑变性(AMD)以脉络膜为特征 新生血管(CNV)：起源于脉络膜的新生血管侵入黄斑。CNV原因 如果不治疗，永久性中心性失明是AMD最威胁视力的病理。现在 应用程序将调查一类新的CNV疗法的有效性和调节机制。反- 血管内皮生长因子疗法是治疗CNV的标准疗法，如果在CNV上进行治疗，可以保护中心视力 无限期复发的基础上，但已达到其治疗门槛。此外，这些疗法非常昂贵。 必须在玻璃体内注射，增加了并发症和患者不依从的风险，并限制了 向受卫生保健提供不平等影响的经济困难患者提供治疗。 本申请有可能识别CNV的新疗法和可用药靶点， 特别是那些可以全身给药而不是玻璃体内给药的药物，因此更容易获得 对服务不足的人群。

ABSTRACT Advanced neovascular age-related macular degeneration (AMD) is characterized by choroidal neovascularization (CNV), in which neovessels originating from the choroid invade the macula. CNV causes permanent central blindness if left untreated and is the most sight-threatening pathology of AMD. The present application will investigate the efficacy and regulatory mechanisms for a new class of CNV therapeutics. Anti- VEGF therapies are the standard of care for CNV and can preserve central vision if administered on an indefinitely recurring basis but have reached their therapeutic threshold. Moreover, these therapies are costly and must be injected intravitreally, increasing the risk of complications and patient noncompliance, and limiting availability of treatment to economically disadvantaged patients affected by disparities in health care delivery. The present application has the potential to identify new therapeutics and druggable targets for CNV, particularly those that can be administered systemically rather than intravitreally, and are thus more accessible to underserved populations.