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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.

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

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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