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Smart-4-Fabry SIGNED

Smart multifunctional GLA-nanoformulation for Fabry disease

Total Cost €

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EC-Contrib. €

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Partnership

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 Smart-4-Fabry project word cloud

Explore the words cloud of the Smart-4-Fabry project. It provides you a very rough idea of what is the project "Smart-4-Fabry" about.

cure    ert    cellular    drawbacks    immunogenicity    stimulate    pd    decline    trl3    serious    cell    nano    poc    transport    exogenously    intravenously    fabry    disorders    fulfillment    strategy    pk    manufacturing    assembly    efficacy    biotechnology    lack    therapies    detriment    constitutes    global    reverse    biological    health    components    experimental    rare    regulatory    liposomal    nanoformulation    vasculopathy    recombinant    free    definitive    group    fd    quality    bbb    instability    cross    glycosphingolipids    cco2    nanotechnology    replacement    kidneys    lsd    materials    galactosidase    barriers    heart    shown    existence    smart    alpha    representing    societal    lysosomal    mainly    nanoformulated    industrial    vivo    preclinical    molecular    deficiency    green    disease    gla    kets    enzyme    excellent    incidence    neutral    diseases    exhibits    administered    storage    samrt    patients    profile    protein    membranes    jobs    gmps    contribution    treatment    final    trl5    nervous    accumulation    therapy   

Project "Smart-4-Fabry" data sheet

The following table provides information about the project.

Coordinator
CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED M.P. 

Organization address
address: CALLE MONFORTE DE LEMOS 5
city: MADRID
postcode: 28029
website: www.ciberisciii.es

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Spain [ES]
 Project website http://smart4fabry.eu/
 Total cost 5˙844˙508 €
 EC max contribution 5˙844˙508 € (100%)
 Programme 1. H2020-EU.2.1.2. (INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies – Nanotechnologies)
 Code Call H2020-NMBP-2016-two-stage
 Funding Scheme RIA
 Starting year 2017
 Duration (year-month-day) from 2017-01-01   to  2020-12-31

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED M.P. ES (MADRID) coordinator 1˙819˙786.00
2    LEANBIO SL ES (AIGUAFREDA) participant 811˙250.00
3    NANOMOL TECHNOLOGIES SL ES (CERDANYOLA DEL VALLES) participant 565˙088.00
4    JOANNEUM RESEARCH FORSCHUNGSGESELLSCHAFT MBH AT (GRAZ) participant 556˙330.00
5    BIOKERALTY RESEARCH INSTITUTE AIE ES (ARCAUTE ALAVA) participant 533˙325.00
6    COVANCE LABORATORIES LTD UK (HARROGATE) participant 434˙395.00
7    TECHNION - ISRAEL INSTITUTE OF TECHNOLOGY IL (HAIFA) participant 393˙705.00
8    AARHUS UNIVERSITET DK (AARHUS C) participant 342˙925.00
9    BIONANONET FORSCHUNGSGESELLSCHAFT MBH AT (GRAZ) participant 205˙078.00
10    DRUG DEVELOPMENT AND REGULATION SL ES (BARCELONA) participant 182˙625.00

Map

 Project 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.

 Deliverables

List of deliverables.
Use and dissemination Plan Documents, reports 2020-02-25 15:22:05
Smart-4-Fabry public website Websites, patent fillings, videos etc. 2020-02-25 15:22:05
Linkedin, Youtube, Twitter Documents, reports 2020-02-25 15:22:05

Take a look to the deliverables list in detail:  detailed list of Smart-4-Fabry deliverables.

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The information about "SMART-4-FABRY" are provided by the European Opendata Portal: CORDIS opendata.

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