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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.

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

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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