A new class of wet AMD therapeutics

摘要

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.