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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.

Field of science

  • /engineering and technology/nanotechnology
  • /engineering and technology/industrial biotechnology
  • /social sciences/social and economic geography/transport
  • /natural sciences/biological sciences/biochemistry/biomolecules/proteins/enzymes

Call for proposal

H2020-NMBP-2016-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

Coordinator

CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED M.P.
Address
Calle Monforte De Lemos 5
28029 Madrid
Spain
Activity type
Research Organisations
EU contribution
€ 1 819 786,13

Participants (9)

BIOKERALTY RESEARCH INSTITUTE AIE
Spain
EU contribution
€ 533 325
Address
Entidad Arcaute, 005, Arcaute
01192 Arcaute Alava
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
NANOMOL TECHNOLOGIES SL
Spain
EU contribution
€ 565 088,75
Address
Lg Edifici Eureka Campus Uab Bellaterra
08193 Cerdanyola Del Valles
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
DRUG DEVELOPMENT AND REGULATION SL
Spain
EU contribution
€ 182 625
Address
C Consell De Cent 375
08009 Barcelona
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
TECHNION - ISRAEL INSTITUTE OF TECHNOLOGY
Israel
EU contribution
€ 393 705
Address
Senate Building Technion City
32000 Haifa
Activity type
Higher or Secondary Education Establishments
AARHUS UNIVERSITET
Denmark
EU contribution
€ 342 925
Address
Nordre Ringgade 1
8000 Aarhus C
Activity type
Higher or Secondary Education Establishments
JOANNEUM RESEARCH FORSCHUNGSGESELLSCHAFT MBH
Austria
EU contribution
€ 556 330
Address
Leonhardstrasse 59
8010 Graz
Activity type
Research Organisations
BIONANONET FORSCHUNGSGESELLSCHAFT MBH
Austria
EU contribution
€ 205 078,75
Address
Steyrergasse 17
8010 Graz
Activity type
Research Organisations
COVANCE LABORATORIES LTD
United Kingdom
EU contribution
€ 434 395
Address
Otley Road
HG3 IPY Harrogate
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
LEANBIO SL
Spain
EU contribution
€ 811 250
Address
Cl Marinada Num 32
08591 Aiguafreda
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)