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Fabry disease awareness month, April

The Fabry International Network (FIN) association established the month of April as the “month of Fabry” to raise awareness and educate about this disease, a rare, progressive and with multi-organ involvement pathology.

Fabry Disease is one of several dozen Lysosomal Storage Disorders that interfere with the body’s ability to break down specific fatty substances. It is a rare disease and because the rate of occurrence is less than 1 in 200,000, it is considered as one of the many “Orphan” diseases. It is more common in women, but it occurs with greater severity in men.

Fabry disease is a metabolic disease that is produced by a deficiency of the ysosomal enzyme Alpha galactosidase. It is transmitted on the X chromosome. Fabry affected patients are missing alpha-galactosidase A (alpha-gal A) which results in sugars and fatty acids (Gb3) accumulating in the cells throughout the body and impairs the function of several major organs including the kidneys and heart. In 2001, enzyme replacement therapy appeared when the alpha-galactosidase protein (alpha- and beta-agalsidase) was synthesized in the laboratory using genetic engineering techniques. This treatment is injected into patients every 15 days to replenish the deficit levels of this enzyme and stop the progression of the disease.

CIBER-BBN, partner of NANBIOSIS, leads the European project Smart4fabry funded by the Horizon 2020 program, which will be developed through a consortium formed by 14 partners from 5 different countries. The CIBER-BBN coordinates the project through the participation of four of its groups that coordinate four units of NANBIOSIS (U1.Protein Production Platform (PPP), U3. Synthesis of Peptides Unit, U6. Biomaterial Processing and Nanostructuring Unit and U20. In Vivo Experimental Platform.) In addition, the consortium is formed by the University of Aarhus (Denmark), Technion Israel Institute of Technology (Israel), Joanneum Research (Austria), Biopraxis Research AIE (Spain), the spin off Nanomol Technologies SL (Spain) ), BioNanoNet (Austria), Drug Development and Regulation SL (Spain), the Covance Laboratories LTD group (UK), and Leanbio SL (Spain) Smart-4-Fabry has been conceived and developed to obtain a new nanoformulation of GLA, that will improve the efficacy and toleration of the treatment with non-formulated GLA. The final benefit will be seen as a considerable reduction on the Fabry disease treatment cost and a substantial improvement in the life-quality of Fabry disease patients.

Fabry International Network, FIN was established in 2005, as a non-for-profit organization registered in The Netherlands. The primary aim of the project is to facilitate collaboration between patient organizations around the world to support those affected by Fabry disease

FIN is connected to over 45 countries around the world. Membership is free and open to any National Patient Organization in which Fabry patients are represented. The National Fabry Disease Foundation – USA, for April 2018 Fabry Disease Awareness Month, have been providing an educational or information post on their Facebook page, every day of the month in April. The NFDF also distributed their My Health Handbook kit  and, so far, distributed about 700 kits to individuals with Fabry disease. Fabry Australia have a new website and they are also running a new social media campaign. Fabry Support & Informatie Groep Nederland, FSIGN, since 2005  has organized every first Saturday of April (in the Fabry Awareness Month April) to be the Fabry women’s day. Japan Fabry Disease Patients and Family Association, in awareness month JFA held an open seminar at Fukuoka University Medical hall with lectures on three major topics: Newborn Mass Screening, Current Treatments and Employment and Clinical Genomics. In Spain the Fabry patient organization are the Spanish Fabry MPS Association

 

The Fabry International Network will cellebrate the 6th Fabry Expert Meeting on
8th – 10th June 2018 at the Vilnius Grand Resort, Ežeraičių g. 2, Ežeraičių km., Avižienių sen., Vilniaus raj., LT-14200, Lietuva.

DRAFT Full Program

 

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Extraordinary doctoral award

Ingrid Cabrera, member of the NANOMOL group that coordinates Unit 6 of NANBIOSIS, has obtained the extraordinary doctoral award from the Universitat Autònoma de Barcelona for her thesis “Nanovesicle-bioactive conjugates to be used as nanomedicines, prepared by a one-step scalable method Using CO₂-expanded solvents“.

This thesis was directed by Jaume Veciana, Scientific Director of NANBIOSIS and Nora Ventosa, Scientific Director of Unit 6 of NANBIOSIS.

The main objectives of the  PhD Thesis of Dr. Ingrid Cabrera were the preparation and study of multifunctional nanovesicle based nanoformulation of alfa-galactosidase for the treatment of Fabry desease as well as of another multifunctional nanovesicle based nanoformulation of epidermal growth factor for the treatment of complex wounds. Both nanoformulations were prepared by a one-step procedure using compressed fluids at U6 of Nanbiosis. This platform also provided many nanoparticles characterization facilities that enabled to obtain important information on the supramolecular organization and characteristics of the produced multifunctional nanovesicles that were key for understanding their biological activities.

Extraordinary doctoral award
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Unit 1 of NANBIOSIS, Protein Production Platform (PPP), Unit 3, Synthesis of Peptides, Unit 20, In Vivo Experimental Platform and Unit 6, Biomaterial Processing and Nanostructuring Unit, have jointly developed the research conducted in relation with a CO2-based methodology for the one-step production of protein-nanoliposome conjugates as bio-active nanomaterials with therapeutic interest. The results have been published in Advanced Healthcare Materials: http://www.ncbi.nlm.nih.gov/pubmed/26890358

Unit 1 of NANBIOSIS, Protein Production Platform (PPP), Unit 3, Synthesis of Peptides, Unit 20, In Vivo Experimental Platform and Unit 6, Biomaterial Processing and Nanostructuring Unit, have jointly developed the research conducted in relation with a CO2-based methodology for the one-step production of protein-nanoliposome conjugates as bio-active nanomaterials with therapeutic interest. The results have been published in Advanced Healthcare Materials: http://www.ncbi.nlm.nih.gov/pubmed/26890358

“a-Galactosidase A Loaded Nanoliposomes with Enhanced Enzymatic Activity and Intracellular Penetration I. Cabrera, I. Abasolo, J. L. Corchero, E. Elizondo,  P. Rivera, E. Moreno, J. Faraudo, S. Sala, D. Bueno, E. González-Mira, M. Rivas, M. Melgarejo, D. Pulido, F. Albericio, M. Royo, A. Villaverde, M. F. García-Parajo, S. Schwartz Jr., N. Ventosa,*, and J. Veciana,*

Lysosomal storage disorders (LSD) are caused by lysosomal dysfunction usually as a consequence of deficiency of a single enzyme required for the metabolism of macromolecules such as lipids, glycoproteins and mucopolysaccharides. For instance, the lack of alpha-Galactosidase A (GLA) activity in Fabry disease patients causes the accumulation of glycosphingolipids in the vasculature leading to multiple organ pathology.

Enzyme replacement therapy (ERT), which is the most common treatment of LSD, exhibits several drawbacks mainly related to the instability and low efficacy of the exogenously administered therapeutic enzyme. In this work, the unprecedented increased enzymatic activity and intracellular penetration achieved by the association of a human recombinant GLA to nanoliposomes functionalized with RGD peptides is reported. Moreover, these new GLA loaded nanoliposomes lead to a higher efficacy in the reduction of the GLA substrate named globotriasylceramide (Gb3) in a cellular model of Fabry disease, than that achieved by the same concentration of the free enzyme. The preparation of these new liposomal formulations by DELOS-SUSP, based on the Depressurization of a CO2-Expanded Liquid Organic Solution, shows the great potential of this CO2-based methodology for the one-step production of protein-nanoliposome conjugates as bioactive nanomaterials with therapeutic interest.

“a-Galactosidase A Loaded Nanoliposomes with Enhanced Enzymatic Activity and Intracellular Penetration”
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