RDCVF
Rod-derived Cone Viability Factor
| Coordinatore | INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
Organization address
address: 101 Rue de Tolbiac contact info |
| Nazionalità Coordinatore | France [FR] |
| Sito del progetto | http://www.rdcvf.eu/ |
| Totale costo | 3˙934˙701 € |
| EC contributo | 2˙623˙333 € |
| Programma | FP7-HEALTH
Specific Programme "Cooperation": Health |
| Code Call | FP7-HEALTH-2009-single-stage |
| Funding Scheme | CP-FP |
| Anno di inizio | 2010 |
| Periodo (anno-mese-giorno) | 2010-03-01 - 2013-02-28 |
Partecipanti
| # | ||||
|---|---|---|---|---|
| 1 |
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
Organization address
address: 101 Rue de Tolbiac contact info |
FR (PARIS) | coordinator | 553˙750.00 |
| 2 |
FOVEA PHARMACEUTICALS
Organization address
address: Institut de la Vision - rue Moreau 17 contact info |
FR (France) | participant | 755˙333.00 |
| 3 |
EBERHARD KARLS UNIVERSITAET TUEBINGEN
Organization address
address: GESCHWISTER-SCHOLL-PLATZ contact info |
DE (TUEBINGEN) | participant | 601˙600.00 |
| 4 |
UNIVERSIDADE DE COIMBRA
Organization address
address: PACO DAS ESCOLAS contact info |
PT (COIMBRA) | participant | 500˙750.00 |
| 5 |
FONDATION DE COOPERATION SCIENTIFIQUE VOIR ET ENTENDRE
Organization address
address: rue de Charenton 28 contact info |
FR (PARIS) | participant | 211˙900.00 |
Mappa
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Obiettivo del progetto (Objective)
'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.'