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Novel advanced and repurposed therapeutics for vision restoration in a group of severe rare ocular surface diseases: from validation to first clinical investigations

Project description

New hope for rare eye diseases

Millions of people in Europe suffer from rare eye diseases that can cause severe vision loss or blindness. Many of these conditions damage the cornea and the surface of the eye. Existing treatments are often expensive, ineffective, and come with serious side effects. Better solutions are needed. In this context, the EU-funded RESTORE VISION project is working to change this situation. Scientists, small businesses, and patient representatives have joined forces to develop better treatments for seven rare eye diseases. They are repurposing six existing drugs and creating three new ones, all designed to restore eye health. Overall, the project aims to bring hope to over 500 000 patients across Europe.

Objective

Rare Eye Diseases (REDs) are a major cause of visual impairment and blindness in Europe, affecting patients of all ages. The RESTORE VISION Consortium identified a group of 7 REDs all affecting the cornea and ocular surface that cause severe vision impairment and blindness and have inadequate treatment options today. Onset and progression is characterised by overlapping common pathophysiologic mechanisms: defective corneal wound healing, nerve degeneration, stem-cell dysfunction and aberrant vessel ingrowth. The 7 REDs targeted are Aniridia-Associated Keratopathy, Neurotrophic Keratopathy, Limbal Stem Cell Deficiency, Ocular Cicatricial Pemphigoid, EEC Syndrome, Ocular Graft versus Host Disease and Corneal Neovascularisation, affecting over 500k patients in Europe.
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. RESTORE VISION brings together actors from across the full value chain: 6 leading research institutions, 3 SMEs and a European patient organisation. We 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.
New therapeutics with game-changing potential will be evaluated for the first time. Our pioneering ‘streams’ approach is based on the repurposing of 6 existing drugs and the development of 3 new compounds, all with solid preliminary data showing remarkable effects in restoring the cell physiology, immune, avascular, neural and signalling environment in the cornea. This innovative approach shortcircuits lengthy and complex regulatory and drug development processes, ensuring rapid translation into the clinic.

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Programme(s)

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Topic(s)

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HORIZON-RIA - HORIZON Research and Innovation Actions

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Call for proposal

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(opens in new window) HORIZON-HLTH-2022-DISEASE-06-two-stage

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Coordinator

UNIVERSITY OF GALWAY
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 1 003 836,25
Address
UNIVERSITY ROAD
H91 Galway
Ireland

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Region
Ireland Northern and Western West
Activity type
Higher or Secondary Education Establishments
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Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

€ 1 003 836,25

Participants (9)

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