Objective Dysregulation of cGMP is a pathological hallmark of inherited retinal degenerations (RD) affecting photoreceptors, the sensory cells of the retina. These RDs, including Retinitis Pigmentosa, Lebers Congenital Amaurosis, and Achromatopsia, are major causes of blindness, affecting approximately one in every 2000 individuals worldwide, and remain without effective treatment. In photoreceptors, cGMP is produced by retinal guanylyl cyclase (GC). The two main cGMP targets are cyclic nucleotide gated ion channels (CNGC) and cGMP-dependent protein kinase (PKG). Since attenuation of PKG and CNGC activity can reduce photoreceptor cell death, both proteins constitute potential targets to prevent RD. Recent data suggest that blocking retinal GC may also constitute a viable therapeutic approach.This consortium will study and develop targeted compounds and delivery systems aimed at preventing photoreceptor damage in preclinical disease models. Towards this goal, two SMEs have teamed up with three academic research groups focused on retinal degeneration: the German company BIOLOG specializes on development of cyclic nucleotide based drugs targeting PKG, CNGC, and GC; the Dutch company to-BBB develops systems to deliver drugs across the blood brain/retinal barrier (BBB, BRB, resp.); the groups of V. Marigo (Modena, Italy), P. Ekström (Lund, Sweden), and F. Paquet-Durand (Tübingen, Germany) have a strong and joint collaborative track record of studying photoreceptor degenerative mechanisms as well as on testing and evaluating drug treatment effects. Manufacturing of the most promising drugs fitted to a suitable delivery system will be scaled up towards clinical-size batches and studied towards efficacy, toxicology and off-target effects in model animals. The results of the project will allow the SMEs to further co-develop these drugs towards translation into clinical studies, addressing the high needs of RD patients and the high economic benefit of such therapies. Fields of science natural sciencesbiological sciencesbiochemistrybiomoleculesproteinsmedical and health sciencesclinical medicineophthalmologynatural sciencesbiological sciencesgeneticsnucleotidesmedical and health sciencesbasic medicinetoxicology Programme(s) FP7-HEALTH - Specific Programme "Cooperation": Health Topic(s) HEALTH.2012.2.4.5-1 - Technological approaches to combating sensory impairments Call for proposal FP7-HEALTH-2012-INNOVATION-2 See other projects for this call Funding Scheme CP-FP - Small or medium-scale focused research project Coordinator EBERHARD KARLS UNIVERSITAET TUEBINGEN Address Geschwister-scholl-platz 72074 Tuebingen Germany See on map Region Baden-Württemberg Tübingen Tübingen, Landkreis Activity type Higher or Secondary Education Establishments Administrative Contact Thomas Wheeler-Schilling (Dr.) Links Contact the organisation Opens in new window Website Opens in new window EU contribution € 992 813,74 Participants (5) Sort alphabetically Sort by EU Contribution Expand all Collapse all TO-BBB TECHNOLOGIES BV Participation ended Netherlands EU contribution € 719 117,27 Address Jh oortweg 19 2333 CH Leiden See on map Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Pieter Gaillard (Dr.) Links Contact the organisation Opens in new window Website Opens in new window BIOLOG LSI FORSCHUNGS- UND BETEILIGUNGS-GMBH Germany EU contribution € 1 396 647,48 Address Flughafendamm 9a 28199 Bremen See on map Region Bremen Bremen Bremen, Kreisfreie Stadt Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Hans-Gottfried Genieser (Dr.) Links Contact the organisation Opens in new window Website Opens in new window UNIVERSITA DEGLI STUDI DI MODENA E REGGIO EMILIA Italy EU contribution € 760 292,52 Address Via universita 4 41121 Modena See on map Region Nord-Est Emilia-Romagna Modena Activity type Higher or Secondary Education Establishments Administrative Contact Fabio Prati (Prof.) Links Contact the organisation Opens in new window Website Opens in new window MAX IV Laboratory, Lund University Sweden EU contribution € 766 650,64 Address Paradisgatan 5c 22100 Lund See on map Region Södra Sverige Sydsverige Skåne län Activity type Higher or Secondary Education Establishments Administrative Contact Per Ekström (Dr.) Links Contact the organisation Opens in new window Website Opens in new window SP PROCESS DEVELOPMENT AB Sweden EU contribution € 335 906,35 Address Po box 857 501 15 Boras See on map Region Södra Sverige Västsverige Västra Götalands län Activity type Research Organisations Administrative Contact Christine Svärd (Ms.) Links Contact the organisation Opens in new window Website Opens in new window
