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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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Outcomes and
results

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.

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

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. 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.	ES (MADRID)	coordinator	1˙819˙786.00
2	LEANBIO SL LEANBIO SL Organization address address: CL MARINADA NUM 32 city: AIGUAFREDA postcode: 8591 website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (AIGUAFREDA)	participant	811˙250.00
3	NANOMOL TECHNOLOGIES SL NANOMOL TECHNOLOGIES SL Organization address address: LG EDIFICI EUREKA CAMPUS UAB BELLATERRA city: CERDANYOLA DEL VALLES postcode: 8193 website: www.nanomol-tech.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (CERDANYOLA DEL VALLES)	participant	565˙088.00
4	JOANNEUM RESEARCH FORSCHUNGSGESELLSCHAFT MBH JOANNEUM RESEARCH FORSCHUNGSGESELLSCHAFT MBH Organization address address: LEONHARDSTRASSE 59 city: GRAZ postcode: 8010 website: www.joanneum.at contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	AT (GRAZ)	participant	556˙330.00
5	BIOKERALTY RESEARCH INSTITUTE AIE BIOKERALTY RESEARCH INSTITUTE AIE Organization address address: ENTIDAD ARCAUTE, 005, ARCAUTE city: ARCAUTE ALAVA postcode: 1192 website: www.grupo-praxis.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (ARCAUTE ALAVA)	participant	533˙325.00
6	COVANCE LABORATORIES LTD COVANCE LABORATORIES LTD Organization address address: OTLEY ROAD city: HARROGATE postcode: HG3 IPY website: www.covance.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	UK (HARROGATE)	participant	434˙395.00
7	TECHNION - ISRAEL INSTITUTE OF TECHNOLOGY TECHNION - ISRAEL INSTITUTE OF TECHNOLOGY Organization address address: SENATE BUILDING TECHNION CITY city: HAIFA postcode: 32000 website: www.technion.ac.il contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	IL (HAIFA)	participant	393˙705.00
8	AARHUS UNIVERSITET AARHUS UNIVERSITET Organization address address: NORDRE RINGGADE 1 city: AARHUS C postcode: 8000 website: www.au.dk contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	DK (AARHUS C)	participant	342˙925.00
9	BIONANONET FORSCHUNGSGESELLSCHAFT MBH BIONANONET FORSCHUNGSGESELLSCHAFT MBH Organization address address: STEYRERGASSE 17 city: GRAZ postcode: 8010 website: www.bionanonet.at contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	AT (GRAZ)	participant	205˙078.00
10	DRUG DEVELOPMENT AND REGULATION SL DRUG DEVELOPMENT AND REGULATION SL Organization address address: C CONSELL DE CENT 375 city: BARCELONA postcode: 8009 website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	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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