Objective
The GeneVision proof-of-concept project spins from a major discovery made by the principle investigator (PI) within the RetGeneTx ERC project demonstrating that dual AAV vectors expand AAV DNA transfer capacity in the retina thus allowing delivery of genes whose size cannot be packaged in single AAVs. Thus, dual AAV vectors allow gene therapy of those inherited blinding conditions, like Usher syndrome type Ib (USHIB), Stargardt disease or other forms of retinitis pigmentosa, due to mutations in large genes from which the AAV platform, the best to date for in vivo gene therapy, was so far precluded.
GeneVision’s objective is to bring this initial proof-of-concept in animal models up to commercialization for one retinal application, USHIB, which is particularly severe and early onset. By exploiting both the Orphan Drug Designation obtained from the European Medicina Agency and two patent applications based on data generated by the PI during its consolidator ERC grant, GeneVision will support the early phase clinical development to bring this very innovative gene therapy platform for retinitis pigmentosa up to commercialization. To do so the project plans to validate the business plan already outlined in the proposal, and seek seed funding to create a start-up company which would engage in all activities required to develop a phase I/II trial. If this is successful, the longer plan is to obtain two additional rounds of funding to support a pivotal trial to complete the data required to obtain market authorization and start commercialization. . The success of this project will lay the foundations for the development of therapies for other retinal and non-retinal diseases using dual AAV vectors.
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.
- medical and health sciences medical biotechnology genetic engineering gene therapy
- natural sciences biological sciences genetics DNA
- medical and health sciences clinical medicine ophthalmology
- natural sciences biological sciences genetics mutation
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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.
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H2020-EU.1.1. - EXCELLENT SCIENCE - European Research Council (ERC)
MAIN PROGRAMME
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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.
ERC-POC - Proof of Concept Grant
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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-2016-PoC
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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.
00185 ROMA
Italy
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.