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Financement de l’UE (5 844 509 €) : Smart multifunctional GLA-nanoformulation for Fabry disease Hor29/11/2016 Programme de recherche et d'innovation de l'UE « Horizon »
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Texte
Smart multifunctional GLA-nanoformulation for Fabry disease
Lysosomal storage disorders (LSD) diseases are a group of rare diseases that currently lack a definitive cure. LSD incidence is about 1:5,000 - 1:10,000, representing a serious global health problem. In the case of Fabry LSD Disease (FD), the deficiency in α-Galactosidase A (GLA) enzyme activity results in the cellular accumulation of neutral glycosphingolipids, leading to widespread vasculopathy with particular detriment to the kidneys, heart and nervous system. The current treatment for FD is the Enzyme Replacement Therapy (ERT), in which free GLA recombinant protein is administered intravenously to patients. ERT exhibits several drawbacks mainly related to the instability, high immunogenicity and low efficacy of the exogenously administered GLA to cross biological barriers, such as cell membranes and BBB.The aim of Samrt-4-Fabry project is to achieve excellent quality control over the assembly of the different molecular components of a new liposomal nanoformulation of GLA, nano-GLA, for the treatment of Fabry disease. Nanoformulated GLA has already shown to have better PK/PD profile than free GLA and higher efficacy in vivo. Smart-4-Fabry project will advance nano-GLA from an experimental PoC (TRL3) to preclinical regulatory phase (TRL5-6). A one-step method based on the use of green cCO2, will be used for the manufacturing of this novel nanoformulation under GMPs. The final GLA nanoformulation will have tailored transport of GLA through cell membranes and BBB. Fulfillment of Smart-4-Fabry will impact on a major health problem, the existence of new therapies for rare diseases, which constitutes a priority societal challenge as shown in the H2020 Work Programmes. Another important impact is related to its contribution to support the European Strategy for KETs, which aims to reverse the decline in manufacturing as this will stimulate growth and jobs. Smart-4-Fabry is strongly focusing on three KETs: nanotechnology, industrial biotechnology and advanced materials.
| Aarhus Universitet | 342 925 € |
| Agencia Estatal Consejo Superior de Investigaciones Cientificas | 0,00 € |
| Biokeralty Research Institute AIE | 533 325 € |
| Bionanonet Forschungsgesellschaft mbH | 205 079 € |
| Consorcio Centro de Investigacion Biomedica en Red M.P | 1 819 786 € |
| Drug Development And Regulation SL | 182 625 € |
| Fundacio Hospital Universitari Vall D'Hebron - Institut de Recerca | 0,00 € |
| Fundacio Parc Cientific de Barcelona | 0,00 € |
| Joanneum Research Forschungsgesellschaft mbH | 556 330 € |
| Labcorp Early Development Laboratories Ltd. | 434 395 € |
| Leanbio SL | 811 250 € |
| Nanomol Technologies SL | 565 089 € |
| TECHNION - ISRAEL INSTITUTE OF TECHNOLOGY | 393 705 € |
https://cordis.europa.eu/project/id/720942
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