RESTORE VISION: Novel advanced and repurposed therapeutics for vision restoration in a group of severe rare ocular diseases – Short introductory talk

Abstract

Rare Eye Diseases (REDs) collectively represent a major cause of visual impairment and blindness for children and young adults in Europe. But importantly, REDs also affect adults and the aging population. Defective wound healing at the cornea and ocular surface, excessive inflammation, nerve degeneration, stem cell dysfunction, and aberrant vessel ingrowth are the common denominators in many REDs, representing a critical medical problem and an area of unmet medical need. The onset and progression of many REDs are characterized by common pathophysiologic mechanisms. The goal of many research groups is to disrupt these aberrant mechanisms with the aim of making therapy more effective. Current management is often prohibitively expensive, has low efficacy and leads to debilitating side effects, pointing to a critical medical problem and area of unmet medical need.Here, we will briefly introduce the RESTORE VISION project, which has been recently funded by the European Commission and brings together actors from across the full value chain: 6 leading research institutions, 3 SMEs and a European patient organization. The project will take a ground‐breaking approach to improve eye health by verifying disease pathomechanisms, using cutting‐edge models for each rare disease to test novel and repurposed compounds and determine drug mechanisms of action, formulating compounds as safe eye drop suspensions or subconjunctival drugs, and performing several first‐in‐human trials of novel therapies.