Sparing gene therapy for Inherited ROd COne dystrophies

Descripción del proyecto

Terapia novedosa para la retinosis pigmentaria

La retinosis pigmentosa (RP) es una enfermedad genética que se caracteriza por la degeneración y la pérdida de fotorreceptores en la retina. Los síntomas habituales incluyen la dificultad para ver por la noche y la pérdida de visión periférica. Se trata de una afección heredada que resulta de los cambios en cualquier gen de un grupo de sesenta y cinco. El principal factor que conduce a la ceguera es la pérdida de una proteína descubierta hace poco, el factor de viabilidad de conos derivado de bastones (RdCVF, por sus siglas en inglés), que estimuló la supervivencia de los fotorreceptores en un modelo de ratones de degeneración de la retina. Tiene un enorme potencial terapéutico para una amplia gama de enfermedades degenerativas de la retina incurables. El proyecto SIROCO, financiado con fondos europeos, tiene por objeto desarrollar un tratamiento innovador para la RP, basado en la introducción de RdCVF como factor fotorreceptor neurotrófico clave. El objetivo final es preparar un tratamiento para la retina que consiste en una sola inyección del producto para tratar todas las formas genéticas de la enfermedad. La prueba de concepto se obtuvo en el modelo de ratones de la RP y, en estos momentos, SIROCO está finalizando la validación clínica de su tecnología.

Objetivo

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.