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Smart multifunctional GLA-nanoformulation for Fabry disease

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.

Call for proposal

H2020-NMBP-2016-2017

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Sub call

H2020-NMBP-2016-two-stage

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

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Region
Comunidad de Madrid Comunidad de Madrid Madrid
Activity type
Research Organisations
Non-EU contribution
€ 0,00

Participants (12)

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

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Region
Comunidad de Madrid Comunidad de Madrid Madrid
Activity type
Research Organisations
Non-EU contribution
€ 0,00
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
Non-EU contribution
€ 0,00
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

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Region
Este Cataluña Barcelona
Activity type
Research Organisations
Non-EU contribution
€ 0,00
BIOKERALTY RESEARCH INSTITUTE AIE
Spain
Net EU contribution
€ 533 325,00
Address
Entidad Arcaute 005 Arcaute
01192 Arcaute Alava

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Region
Noreste País Vasco Araba/Álava
Activity type
Other
Non-EU contribution
€ 0,00
NANOMOL TECHNOLOGIES SL
Spain
Net EU contribution
€ 565 088,75
Address
Lg Edifici Eureka Campus Uab Bellaterra
08193 Cerdanyola Del Valles

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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)
Non-EU contribution
€ 0,00
DRUG DEVELOPMENT AND REGULATION SL
Spain
Net EU contribution
€ 182 625,00
Address
C Consell De Cent 375
08009 Barcelona

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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)
Non-EU contribution
€ 0,00
TECHNION - ISRAEL INSTITUTE OF TECHNOLOGY
Israel
Net EU contribution
€ 393 705,00
Address
Senate Building Technion City
32000 Haifa

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Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
AARHUS UNIVERSITET
Denmark
Net EU contribution
€ 342 925,00
Address
Nordre Ringgade 1
8000 Aarhus C

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Region
Danmark Midtjylland Østjylland
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
JOANNEUM RESEARCH FORSCHUNGSGESELLSCHAFT MBH
Austria
Net EU contribution
€ 556 330,00
Address
Leonhardstrasse 59
8010 Graz

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Region
Südösterreich Steiermark Graz
Activity type
Research Organisations
Non-EU contribution
€ 0,00
BIONANONET FORSCHUNGSGESELLSCHAFT MBH
Austria
Net EU contribution
€ 205 078,75
Address
Kaiser-josef-platz 9
8010 Graz

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Region
Südösterreich Steiermark Graz
Activity type
Research Organisations
Non-EU contribution
€ 0,00
LABCORP EARLY DEVELOPMENT LABORATORIES LIMITED
United Kingdom
Net EU contribution
€ 434 395,00
Address
Otley Road
HG3 IPY Harrogate

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Region
Yorkshire and the Humber North Yorkshire North Yorkshire CC
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
LEANBIO SL
Spain
Net EU contribution
€ 811 250,00
Address
Cl Marinada Num 32
08591 Aiguafreda

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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)
Non-EU contribution
€ 0,00