Cholesterol Removal as a Treatment for Dry Age-Related Macular Degeneration

Abstract The leading cause of blindness in people over 50 in industrialized nations including the United States is age-related macular degeneration (AMD). Early AMD is characterized by cholesterol accumulation under the neurosensory retina called sub-retinal drusenoid deposits or under the retinal pigment epithelium called drusen. Vision loss occurs in advanced AMD, with “wet” AMD comprising 20% of cases driven by angiogenesis, or from “dry” AMD (80% of cases) driven by cholesterol accumulation and accompanying cell death (geographic atrophy). Mobius has an expert team in cholesterol biology, lipid transport in the eye, lipoprotein nanoparticle (nanodisc) production, and human therapeutics to tackle cholesterol removal from the eye. The Phase 1 goal is to demonstrate the feasibility of optimized nanodisc technology to facilitate cholesterol removal, and demonstrate therapeutic potential to treat cholesterol accumulation to treat dry AMD. The aims of Phase 1 will be to initially design the optimal prototype nanodisc for cholesterol removal, and then test that prototype in cells and pre-clinical models of relevance in AMD.