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VHIR participates in a European project against Fabry disease

The Drug Delivery and Targeting group from the CIBBIM-Nanomedicine at VHIR, led by Dr. Simó Schwartz Jr., participates in a new European project, which aims the innovation and improvement in the treatment for Fabry disease, a rare condition X-linked which affects 1 in 7.000 people approximately. The project is awarded with 5,8 milion in the Horizon 2020 program of the European Comission, and involves the participation of 14 institutions from academia and industry.

Fabry disease belongs to the lisosomal disorders group and it is characterized by the deficiency of the enzymatic activity of α-galactosidase A. The lack of this enzyme causes cell damage which affects the skin, the kidneys, the heart and the central nervous system, among others. Nowadays, the treatment for Fabry disease consists in the administration of the protein which the patients of Fabry disease do not have.

So as to improve the treatment against this rare disease, the VHIR investigators performed several previous studies which have led them to participate in this European Project. Specifically, they participate in the NanoFabry project, awarded by La Marató de TV3, the Lipocell project and Terarmet, a project awarded by the Ministerio de Economía y Competitividad. Dr. Ibane Abasolo, principal investigator of the SMART-4-FABRY in the CIBBIM group, explains that “with this project we will work in the possibility of crossing the blood-brain barrier, something that is crucial for the treatment of the lisosomal diseases and that could benefit other rare conditions with neurological affectation”.

Moreover, the advisory board of the SMART-4-FABRY involves the participation of Dr. Jordi Pérez, physician of the minor diseases unit of the Hospital Vall d’Hebron and Dr. Mari Carmen Domínguez, member of the CIBBIM-Nanomedicina VHIR group.

Dr. Abasolo assures that thanks to the participation of the VHIR and the rest of groups in this European project, “we will be ready to start the development of an innovative treatment, more beneficial for the patients”.

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Inano medarbejdere

Prof. Jan Skov Pedersen, Department of Chemistry and iNANO, is part of the new Horizon 2020 Framework Programme project “Smart-4-Fabry” (Smart multifunctional GLA-nanoformulation).

The project is funded under the action ‘H2020-EU.2.1.2. – INDUSTRIAL LEADERSHIP – Leadership in enabling and industrial technologies –  Nanotechnologies’. The project started on 1st January 2017, and runs for 4 years with 10 partners and a budget of 5,800,000 € of which 343,000 is allocated for Aarhus University.

The research of Smart-4-Fabry project concerns development of drug delivery systems based on liposomes for treatment of Fabry disease. Advanced formulation and characterization techniques will be used for achieving excellent quality control over the assembly of the different molecular components of a new liposomal nano-formulation which contains the α-Galactosidase A (GLA) enzyme as the active component. Fabry disease belongs to a group of lysosomal storage disorders (LSD) diseases that currently lack a definitive cure. In the case of Fabry LSD Disease, the deficiency in GLA activity results in the cellular accumulation of neutral glycosphingolipids, leading to widespread blood vessel disorder with particular detriment to the kidneys, heart and nerve system.

Inano/

proyectos de convocatorias competitivas

Los trastornos de almacenamiento lisosomal (LSD) son un grupo de enfermedades raras que en la actualidad carecen de una cura definitiva. En el caso de la enfermedad de Fabry, existe una deficiencia en α-galactosidasa A (GLA). Como resultado de la inexistencia de la enzima se produce una acumulación celular de glicoesfingolípidos neutros, que conduce a la vasculopatía generalizada con especial perjuicio de los riñones, el corazón y del sistema nervioso. El tratamiento actual para la FD es la terapia de sustitución enzimática (ERT) en la que proteína recombinante GLA libre se administra por vía intravenosa a los pacientes.

Este tipo de tratamientos presenta varias desventajas relacionadas principalmente con la inestabilidad, alta inmunogenicidad y baja eficacia de la GLA exógenamente administrada, así como incapacidad de la misma de cruzar barreras biológicas tales como la barrera hematoencefálica.

El objetivo del proyecto Smart-4-Fabry es lograr un excelente control de calidad sobre el conjunto de los diferentes componentes moleculares de una nueva nanoformulación liposomal de GLA (nano-GLA) para el tratamiento de la enfermedad de Fabry. Esta nano-GLA ya se ha demostrado que tiene un mejor perfil de PK / PD respecto a la GLA libre así como una mayor eficacia in vivo. El proyecto Smart-4-Fabry avanzará el prototipo de la nano-GLA como prueba de concepto (TRL3) hasta la fase de regulación preclínica (TRL5-6). La formulación final nana-GLA permitirá el transporte a medida del enzima GLA a través de las membranas celulares y de la barrera hematoencefálica.

Smart-4-Fabry tendrá un impacto en un importante problema de salud que es la existencia de nuevas terapias para las enfermedades raras, lo que constituye un reto social prioritario tal y como se muestra en el programa de trabajo H2020 de la comunidad europea. Smart-4Fabry contribuye a la Estrategia Europea para las KETs, que tiene como objetivo revertir la disminución de la fabricación ya que esto estimulará el crecimiento y el empleo. Smart-4-Fabry está fuertemente centrado en tres KETs: nanotecnología, biotecnología industrial y materiales avanzados.

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Nanomol Technologies

Nanomol Technologies is part of the new Horizon 2020 Framework Programme project “Smart-4-Fabry” (Smart multifunctional GLA-nanoformulation for Fabry disease)

The project has started on January 1st, 2017, and will run for 4 years with 10 partners and a budget of 5,800,000 €.

The research of Smart-4-Fabry project concerns development of drug delivery systems based on liposomes for treatment of Fabry disease. Advanced formulation and characterization techniques will be used for achieving excellent quality control over the assembly of the different molecular components of a new liposomal nano-formulation which contains the α-Galactosidase A (GLA) enzyme as the active component. Fabry disease belongs to a group of lysosomal storage disorder (LSD) diseases that currently lack a definitive cure. In the case of Fabry LSD disease, the deficiency in GLA activity results in the cellular accumulation of neutral glycosphingolipids, leading to widespread blood vessel disorder with particular detriment to the kidneys, heart and nervous system.

Nanomol-tech/

Kick-off Meeting

Kick-off Meeting of the Smart-4-Fabry Project will be held in Barcelona from the 16th to the 19th January 2017.