Project description
Novel therapy for retinitis pigmentosa
Retinitis pigmentosa (RP) is a genetic disorder involving breakdown and loss of photoreceptors in the retina. Common symptoms include difficulty seeing at night and a loss of peripheral vision. This is an inherited disorder resulting from changes in any one of 65 genes. The main factor leading to blindness is loss of a recently discovered protein, rod-derived cone viability factor (RdCVF), which stimulated photoreceptor survival in a mouse model of retinal degeneration. It has enormous therapeutic potential for a wide range of untreatable retinal degenerative disorders. The EU-funded SIROCO project aims to develop innovative treatment for RP based on introduction of RdCVF as a key neurotrophic photoreceptor factor. The ultimate goal is to develop a single-injection retinal delivery of the product to treat all genetic forms of the disease. Proof of concept was obtained in the mouse model of RP and SIROCO is currently finalising clinical validation of this technology.
Objective
Many believe that corrective ophthalmology gene therapy is the future for rare diseases such as retinitis pigmentosa (RP), a rare, inherited retinal disorders characterized by diffuse progressive dysfunction of primarily rod photoreceptors, with subsequent degeneration of cone photoreceptors. This degenerative disease causes blindness to young people and adults. But with this kind of therapy only treating 1 out of 65 mutations of the pathology, it means a small group of patents can be targeted.
So, what do the remaining 2 million people have as on option?
SparingVision have developed an innovative treatment that can stop the evolution of this blindness and we call it RdCVF. The first vision saving treatment of its kind.
What makes it different is that in 2004, J Sahel and T Leveillard identified that in RP, the main factor that leads to blindness is a loss of RdCVF with is a neurotrophic factor for the cones (photoreceptor). This means that by a single injection under the retina SparingVision’ product can treat all the genetic forms of RP, potentially treat patients with dry AMD, and provide patients with long lasting efficacy.
In SparingVision’ proof of concept the product was injected to mice models provided almost immediate protection and repair to the cones and the evolution to blindness is stopped. Behind this product is a very strong team, a world renamed scientific advisory board. The product development has been secured with strong IP, exclusive licenses agreements and pursue R&D with research collaboration agreements and raised money.
So, with the help of SME INSTRUMENT supporting the project SIROCO, SparingVision will be able to finalize the next steps of the clinical validation of an unique therapeutic approach and make the product finally accessible and being the solution to stop unnecessary blindness and other degenerative conditions.
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
- medical and health sciences clinical medicine ophthalmology
- natural sciences biological sciences genetics mutation
- medical and health sciences basic medicine pathology
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
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.2.3. - INDUSTRIAL LEADERSHIP - Innovation In SMEs
MAIN PROGRAMME
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H2020-EU.3. - PRIORITY 'Societal challenges
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H2020-EU.2.1. - INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies
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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.
SME-2 - SME instrument phase 2
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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) H2020-EIC-SMEInst-2018-2020
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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.
75012 Paris
France
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
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.