EP4351541A2 — Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders

USPTO Abstract

The present invention relates to agents which inhibit the expression and/or activity of transthyretin (TTR), e.g., a double stranded RNA (dsRNA) agent, or salt thereof, or an antisense oligonucleotide or a gene therapy targeting TTR, and the use of these agents in methods of treating or preventing Startgardt's disease, methods of decreasing Vitamin A levels or formation of toxic Vitamin A metabolites, and/or methods of halting progression of vision loss in a subject. The present invention also relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the retinal binding protein 4 (RBP4) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an RBP4 gene. The invention futher provides the use of RNAi agent targeting RBP4 and/or nucleic acid agents targeting TTR in methods of preventing and treating an RBP4-associated disorder, e.g., an ocular disease, e.g., Stargardt's disease, diabetic retinopathy, age-related macular degeneration (AMD), e.g., dry AMD and wet AMD; or a metabolic disorder, e.g., a disorder of glucose and lipid homeostasis, e.g., insulin resistance associated with type II diabetes, or a cardiovascular disease.