About Restore Vision

In Europe, approximately 30 million people suffer from blindness and visual impairment. Rare Eye Diseases (REDs) are a major cause of visual impairment and blindness affecting patients of all ages.

Currently, many REDs have few treatments available which are often ineffective and cause significant side effects. To tackle this medical need, Restore Vision will create and test new treatments for 7 REDs affecting the cornea and ocular surface:

Aniridia-Associated Keratopathy
Ocular Cicatricial Pemphigoid
Ectrodactyly-ectodermal dysplasia-clefting syndrome
Neurotrophic Keratopathy
Limbal Stem Cell Deficiency
Ocular Graft versus Host Disease
Corneal Neovascularisation

Our approach

Restore Vision will explore new treatments for 7 rare eye diseases by repurposing existing generic drugs, developing new treatments, and conducting pioneering first-in-human studies to ensure their safety and effectiveness.

To achieve that, the team will work in seven different work packages (WPs):

WP1

Project management.

This work package will be responsible for managing the programme and reporting, ensuring timely execution of the work and completion of WPs 2-7.

WP2

Validation of human drug targets of repurposed drugs and novel therapies.

This work package will investigate the impact of proposed experimental/repurposed substances on human tissue and fluid samples of rare eye diseases. This aims to determine how these substances interact with and influence specific genes, proteins, and pathways of interest within the human body.

WP3

Toxicity and formulation of drugs.

This work package will create eye drop or periocular formulations of certain drugs to improve their effectiveness and ease of use for treating eye conditions. Some of these drugs are already approved for other medical uses and will be adapted for ocular treatment, while others are new and still need human testing. The selected drug candidates will undergo various tests, including animal models and pilot clinical trials, to assess their safety, formulation, and pharmacokinetics/dynamics. Approved in vitro alternatives will also be used to reduce animal testing. The main goals are to create innovative eye drop formulations for Restore Vision drugs and ensure their safety for the eyes.

WP4

Evaluating the potential of novel therapeutic drugs in preclinical models for the 7 REDs.

This work package will assess the potential of new therapeutic drugs in laboratory and animal models for the 7 specific REDs (Rare Eye Diseases). These drugs will be evaluated in models that faithfully recapitulate human disease. Initial data has shown positive effects that can be extended to several RED with similar biological processes. The specific goals of WP4 are to obtain suitable models for the REDs, test the therapeutic effectiveness of Restore vision drugs in these models, and explore how these drugs work (Mechanism of Action or MoA).

WP5

First in human pilot clinical studies.

This work package will test the most promising therapeutic drugs in early pilot clinical trials involving patients with REDs. It will evaluate their safety and effectiveness in these trials and coordinate the assessment of compassionate and off-label use of these and other compounds in REDs. The ultimate goal is to identify effective treatments for these rare eye conditions.

WP6

Communications, dissemination and exploitation.

This work package will ensure effective intra-consortium and external communication, maximise project impacts and uptake of results; support effective conversion of outputs into marketable products and engage meaningfully and strategically with key stakeholders, particularly the patients.

WP7

Ethics matters and regulatory affairs.

This work package will provide assistance, coordination, and oversight for the ethical aspects of our studies involving animals and humans. Requirements for regulatory approvals for bringing new drugs into clinical use will also be outlined and followed for a streamlined drug approval process.

The project will innovate by:

1

Validating novel drug targets for 7 rare eye diseases and identifying mechanisms of action (WP2)

2

Generating new drug formulations for 9 novel drugs (WP3)

3

Building a novel in vitro and in vivo preclinical RED testing platform for mesenchymal stem/stromal cells (MSC) derivatives and repurposed drugs that have never been used before in REDs (WP4)

4

Evaluating new molecules that have never been tested or used in humans (WP4)

5

Conducting multiple first-in-human trials (WP5)

Interested in Restore vision?

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