Objetivo The discovery of RdCVF (Rod-derived Cone Viability Factor) has provided a clue to understanding the secondary loss of cone photoreceptors (and of central and light-adapted vision) following the degeneration of rod photoreceptors as a consequence of mutations expressed only in rods in most cases of rod-cone degenerations (or retinitis pigmentosa : RP). In two different rodent models of RP, intraocular administration of RdCVF increased significantly cone survival and function. Given the unparalleled genetic heterogeneity of retinal dystrophies, including RP, the delivery of RdCVF appears as a promising, mutation independent strategy for preserving central vision, even at late stages of the disease, opening a wide window for neuroprotection. RdCVF, discovered by team 1, and developed by team 5, has been granted by the EMEA and FDA the Orphan Status. Reaching the stage of phase I/II trials with RdCVF protein therapy in RP implies several key preclinical milestones: 1) the production of GMP grade proteins and their functional validation in in vitro and in vivo assays, 2) pharmacokinetic and pharmacodynamic studies determining, the dosage, half life, site of injection of the protein, while 3) toxicology studies will be performed in normal and mutant mice and rats, and in monkeys. In parallel, based on the knowledge gained by partner 1 on tryparedoxins (the family of RdCVF) and on RdCVF sequence and paralogs, attempts will be made to 4) optimize the therapeutic protein. In order to reduce the injected dose and to provide a steady level of RdCVF, innovative delivery systems such as nanoparticules will be developed. These steps, conducted by renown academic partners in the fields of neuroprotection and toxicology, experienced industrials and subcontractants, will lead to a proof of safety and concept in advanced RP. This may provide a novel, widely applicable approach to an untreatable blinding condition, while hinting towards extension to other neurodegenerative diseases. Ámbito científico medical and health sciencesmedical biotechnologynatural sciencesbiological sciencesgeneticsmutationmedical and health sciencesclinical medicineophthalmologyretinopathymedical and health sciencesbasic medicinetoxicology Programa(s) FP7-HEALTH - Specific Programme "Cooperation": Health Tema(s) HEALTH-2009-2.4.4-2 - Preclinical development of substances with a clear potential as orphan drugs Convocatoria de propuestas FP7-HEALTH-2009-single-stage Consulte otros proyectos de esta convocatoria Régimen de financiación CP-FP - Small or medium-scale focused research project Coordinador INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE Aportación de la UE € 553 750,00 Dirección RUE DE TOLBIAC 101 75654 Paris Francia Ver en el mapa Región Ile-de-France Ile-de-France Paris Tipo de actividad Research Organisations Contacto administrativo Mihaja Auguste (Ms.) Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Coste total Sin datos Participantes (4) Ordenar alfabéticamente Ordenar por aportación de la UE Ampliar todo Contraer todo EBERHARD KARLS UNIVERSITAET TUEBINGEN Alemania Aportación de la UE € 601 600,00 Dirección GESCHWISTER-SCHOLL-PLATZ 72074 Tuebingen Ver en el mapa Región Baden-Württemberg Tübingen Tübingen, Landkreis Tipo de actividad Higher or Secondary Education Establishments Contacto administrativo Thomas Riehle (Mr.) Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Coste total Sin datos UNIVERSIDADE DE COIMBRA Portugal Aportación de la UE € 500 750,00 Dirección PACO DAS ESCOLAS 3004-531 Coimbra Ver en el mapa Región Continente Centro (PT) Região de Coimbra Tipo de actividad Higher or Secondary Education Establishments Contacto administrativo Maria Helena Gil (Prof.) Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Coste total Sin datos FOVEA PHARMACEUTICALS Francia Aportación de la UE € 755 333,00 Dirección Institut de la Vision - rue Moreau 17 75012 France Ver en el mapa Tipo de actividad Private for-profit entities (excluding Higher or Secondary Education Establishments) Contacto administrativo Bernard Gilly (Mr.) Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Coste total Sin datos FONDATION DE COOPERATION SCIENTIFIQUE VOIR ET ENTENDRE Francia Aportación de la UE € 211 900,00 Dirección 17 RUE MOREAU 75012 Paris Ver en el mapa Región Ile-de-France Ile-de-France Val-de-Marne Tipo de actividad Research Organisations Contacto administrativo Olivier Lorentz (Dr.) Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Coste total Sin datos
