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Virus-free gene therapy for retinal diseases : Date:

Eberhard Karls University of Tübingen - Prof Peer Fischer

In people with retinal disease, the light-sensitive sensory cells in the retina are damaged and, in the worst case, die. © Adobe Stock/ Clement C/peopleimages.com

conceptual period 

Recipient: Eberhard Karls University of Tübingen
Funding: GO-Bio initial conceptual phase 2nd round (01/10/2021 to 30/09/2022, EUR 118,747.99)

The goal of the REVeyeVE research project is to develop an alternative to viral ocular gene therapy and thus prevent patients with hereditary retinal diseases from going blind. In Germany alone, 75,000 people are affected, and in some cases, there is no treatment. The researchers are also aiming to extend the developing therapy to common diseases in which a genetic predisposition increases the risk of vision defects and blindness. These include age-related macular degeneration, which affects around 6 million people in Germany.

In 2018, the first viral gene therapy for an eye condition was approved, making causal treatment for a genetic eye disease possible for the first time. At the same time, however, the potential applications are limited, due to frequent side effects such as inflammatory reactions. In addition, the gene size that can be transported by a virus is limited.

The development of non-viral nanovehicles envisaged as part of the project offers the opportunity to overcome these disadvantages. The nanovehicles are well tolerated and are made from biodegradable materials. They can be magnetically controlled and navigated to their destination in a targeted manner. This targeted navigation offers an absolutely unique selling point and makes injections under the retina unnecessary. The nanovehicles can also be loaded with large genes. The advantages of this novel technology contribute significantly to the improvement of ocular gene therapy and protect more patients from going blind. In the initial phase of the project, the chances of exploiting the project idea will be analysed on the basis of economic and legal considerations. In this way, the project contributes to strengthening the life science innovation landscape in Germany in line with the announcement.

feasibility stage

Recipient: Eberhard Karls University of Tübingen
Funding: GO-Bio initial feasibility phase 2 (01/10/2022 to 30/09/2024, EUR 849,010.84)

In Germany alone, hereditary retinal diseases affects 75,000 people. Treatment of the diseases is only possible in a few cases. In 2018, the first viral gene therapy for an eye condition was approved, making causal treatment possible for the first time - a milestone in the treatment of hereditary retinal diseases. However, the application options are limited, the injection in the eye is complicated and there are often side effects such as chronic inflammatory reactions.

The aim of the REVeyeVe-2 research project is to develop an alternative to viral gene therapy that is easier to administer and does not cause inflammation, thereby preventing people with hereditary retinal diseases from going blind. The research project also aims to treat common diseases such as age-related macular degeneration, where a genetic predisposition increases the risk of blindness. This could help six million affected people in Germany.

The targeted development of non-viral nanovehicles could be the solution. The metallic nanovehicles are well tolerated, biodegradable and guidable to their destination by a magnetic field. This targeted navigation offers an absolute unique selling point and makes injections under the retina unnecessary. In addition, unlike viruses, the nanoparticles can also be loaded with large genes. The advantages of this novel technology contribute significantly to the improvement of ocular gene therapy and protect more patients from going blind. In this project, the feasibility of the therapy in the animal eye will be tested and the foundations for further follow-up funding will be laid by completing the team and specifying the commercialisation plan.