Objective 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. Fields of science engineering and technologyindustrial biotechnologymedical and health sciencesmedical biotechnologynanomedicinemedical and health sciencesbasic medicinepathologymedical and health sciencesbasic medicinetoxicologynatural sciencesbiological sciencesbiochemistrybiomoleculesproteinsenzymes Keywords GLA delivery liposomal nanocarrier lipid self-assembly control Fabry disease GMP green manufacturing process cellular membrane penetration blood-brain-barrier crossing peptide targeting Programme(s) H2020-EU.2.1.2. - INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies – Nanotechnologies Main Programme Topic(s) NMBP-10-2016 - Nanoformulation of biologicals Call for proposal H2020-NMBP-2016-2017 See other projects for this call Sub call H2020-NMBP-2016-two-stage Funding Scheme RIA - Research and Innovation action Coordinator CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED M.P. Net EU contribution € 1 370 589,60 Address CALLE MONFORTE DE LEMOS 5 28029 Madrid Spain See on map Region Comunidad de Madrid Comunidad de Madrid Madrid Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 1 819 786,13 Participants (12) Sort alphabetically Sort by Net EU contribution Expand all Collapse all Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. FUNDACIO PARC CIENTIFIC DE BARCELONA Participation ended Spain Net EU contribution € 0,00 Address BALDIRI I REIXAC 4 08028 Barcelona See on map Region Este Cataluña Barcelona Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost No data Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. AGENCIA ESTATAL CONSEJO SUPERIOR DE INVESTIGACIONES CIENTIFICAS Spain Net EU contribution € 182 895,73 Address CALLE SERRANO 117 28006 Madrid See on map Region Comunidad de Madrid Comunidad de Madrid Madrid Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 182 895,73 Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA Spain Net EU contribution € 266 300,80 Address PASSEIG VALL D HEBRON 119-129 EDIFICIO DE RECERCA 08035 Barcelona See on map Region Este Cataluña Barcelona Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 266 300,80 BIOKERALTY RESEARCH INSTITUTE AIE Spain Net EU contribution € 533 325,00 Address ENTIDAD ARCAUTE 005 ARCAUTE 01192 ARCAUTE ALAVA See on map Region Noreste País Vasco Araba/Álava Activity type Other Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 533 325,00 NANOMOL TECHNOLOGIES SL Spain Net EU contribution € 565 088,75 Address LG EDIFICI EUREKA CAMPUS UAB BELLATERRA 08193 Cerdanyola Del Valles See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Este Cataluña Barcelona Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 565 088,75 DRUG DEVELOPMENT AND REGULATION SL Spain Net EU contribution € 182 625,00 Address C CONSELL DE CENT 375 08009 Barcelona See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Este Cataluña Barcelona Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 182 625,00 TECHNION - ISRAEL INSTITUTE OF TECHNOLOGY Israel Net EU contribution € 393 705,00 Address SENATE BUILDING TECHNION CITY 32000 Haifa See on map Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 393 705,00 AARHUS UNIVERSITET Denmark Net EU contribution € 342 925,00 Address NORDRE RINGGADE 1 8000 Aarhus C See on map Region Danmark Midtjylland Østjylland Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 342 925,00 JOANNEUM RESEARCH FORSCHUNGSGESELLSCHAFT MBH Austria Net EU contribution € 556 330,00 Address LEONHARDSTRASSE 59 8010 Graz See on map Region Südösterreich Steiermark Graz Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 556 330,00 BIONANONET FORSCHUNGSGESELLSCHAFT MBH Austria Net EU contribution € 205 078,75 Address KAISER-JOSEF-PLATZ 9 8010 Graz See on map Region Südösterreich Steiermark Graz Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 205 078,75 LABCORP EARLY DEVELOPMENT LABORATORIES LIMITED United Kingdom Net EU contribution € 434 395,00 Address OTLEY ROAD HG3 IPY Harrogate See on map Region Yorkshire and the Humber North Yorkshire North Yorkshire CC Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 434 395,00 LEANBIO SL Spain Net EU contribution € 811 250,00 Address CL MARINADA NUM 32 08591 Aiguafreda See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Este Cataluña Barcelona Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 811 250,00
