Project description
Pioneering gene therapy to restore sight
Vision loss affects hundreds of millions of people worldwide, and for many with genetic eye diseases, treatment remains out of reach. One such condition, Usher syndrome type 1B, causes deafness, balance problems, and blindness due to mutations in the MYO7A gene. The ERC-funded EuroVision project aims to change that. Building on breakthrough research from previous ERC projects, the team has developed a lentiviral gene therapy capable of delivering healthy copies of MYO7A directly to eye cells. Now entering pre-clinical development, this therapy could become the first single-vector treatment for this devastating condition, offering hope to patients and setting the stage for tackling other inherited eye diseases.
Objective
Sight is the most treasured of our senses, followed by hearing and balance. Moderate to severe vision loss affects more than 216 million people worldwide, with an estimated 36 million people suffering from blindness. Monogenic diseases caused by mutations in genes such as MYO7A are responsible for vision loss in over two million people. As current treatment options for genetic vision loss are limited or non-existent, there is a high clinical need for innovative approaches, such as gene therapy. Usher syndrome type 1B (USH1B) is caused by MYO7A mutations and patients suffer from profound deafness accompanied with loss of balance and vision. In my ERC-funded consolidator grant iHEAR and Proof-of-Concept grant MY(O)SENSES, we showed the first ground-breaking data for efficient gene transfer with lentiviral vectors into inner ear cell types and correction of hearing and balance defects caused by mutated MYO7A. The EuroVision PoC proposal aims to advance these valuable research results towards clinical application by completing pre-clinical development, securing IP rights and commercializing a formulated medicinal product to launch a first-in-human clinical LV gene therapy trial to treat USH1B patients. The EuroVision product will be the first and only single-vector treatment that addresses vision loss due to MYO7A mutations in USH1B patients, which has great potential to significantly improve quality of life for these patients as well as to benefit society through relief of costs associated with healthcare and lost productivity. Moreover, this ground-breaking therapy will lay the basis to address other monogenic eye diseases, in which large genes need to be transferred and for which there is an urgent medical need.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- social sciences economics and business economics production economics productivity
- natural sciences biological sciences genetics mutation
You need to log in or register to use this function
We are sorry... an unexpected error occurred during execution.
You need to be authenticated. Your session might have expired.
Thank you for your feedback. You will soon receive an email to confirm the submission. If you have selected to be notified about the reporting status, you will also be contacted when the reporting status will change.
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
-
HORIZON.1.1 - European Research Council (ERC)
MAIN PROGRAMME
See all projects funded under this programme
Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants
See all projects funded under this funding scheme
Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) ERC-2025-POC
See all projects funded under this callHost institution
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.
30625 Hannover
Germany
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.