R.P 2.4 Novel Gene Delivery Platforms for the Treatment of Ocular Disease

Overview

Many ocular diseases remain untreatable with conventional therapies such as pharmacological drugs; consequently, gene therapy has emerged as a promising and innovative therapeutic approach.  This project aims to validate and commercialize novel gene delivery platforms that enable effective DNA delivery to the eye for the treatment of ocular diseases. Addressing the limitations of current gene therapy approaches and topical eye drops, the study focuses on designing advanced delivery formulations capable of crossing ocular barriers and targeting the posterior segment. Research activities include developing and characterizing multiple non-viral delivery platforms with enhanced neuroprotection, photoreceptor targeting, and safety, initially applied to inherited retinal disorders and designed for extension to other currently untreatable ocular conditions.