CORDIS
EU research results

CORDIS

English EN

Smart multifunctional GLA-nanoformulation for Fabry disease

Project information

Grant agreement ID: 720942

Status

Ongoing project

  • Start date

    1 January 2017

  • End date

    31 December 2020

Funded under:

H2020-EU.2.1.2.

  • Overall budget:

    € 5 844 508,63

  • EU contribution

    € 5 844 508,63

Coordinated by:

CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED M.P.

Spain

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.
Leaflet | Map data © OpenStreetMap contributors, Credit: EC-GISCO, © EuroGeographics for the administrative boundaries

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

NANOMOL TECHNOLOGIES SL

Spain

EU Contribution

€ 565 088,75

DRUG DEVELOPMENT AND REGULATION SL

Spain

EU Contribution

€ 182 625

TECHNION - ISRAEL INSTITUTE OF TECHNOLOGY

Israel

EU Contribution

€ 393 705

AARHUS UNIVERSITET

Denmark

EU Contribution

€ 342 925

JOANNEUM RESEARCH FORSCHUNGSGESELLSCHAFT MBH

Austria

EU Contribution

€ 556 330

BIONANONET FORSCHUNGSGESELLSCHAFT MBH

Austria

EU Contribution

€ 205 078,75

COVANCE LABORATORIES LTD

United Kingdom

EU Contribution

€ 434 395

LEANBIO SL

Spain

EU Contribution

€ 811 250

Project information

Grant agreement ID: 720942

Status

Ongoing project

  • Start date

    1 January 2017

  • End date

    31 December 2020

Funded under:

H2020-EU.2.1.2.

  • Overall budget:

    € 5 844 508,63

  • EU contribution

    € 5 844 508,63

Coordinated by:

CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED M.P.

Spain