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.
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Funding Scheme
SME-2 - SME instrument phase 2Coordinator
75012 Paris
France
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.