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News U3

News U3

A more effective nanomedicine has been developed for the treatment of Fabry rare disease.

28 February: International Rare Disease Day

  • This is one of the major achievements of the European Smart4Fabry project, which is now coming to an end after four years of work.
  • The results have been made possible by nanotechnology and the approach developed could be applied to other drugs in the future.
  • The new drug improves on current treatments and helps reduce costs and improve patients’ quality of life.

Barcelona, 26 February 2021.- The advance of nanomedicine opens up new possibilities in the development of drugs, such as the one recently developed for the rare disease Fabry, with improved efficacy compared to existing authorised treatments.

Thus, the European Smart4Fabry project has come to an end with one of the best results possible: the designation of a new orphan drug by the European Commission and the possibility of making progress in the treatment of Fabry, a rare disease that is estimated to affect approximately 2.6 out of every 10,000 people in the EU.

It is a chronic debilitating disease due to recurrent episodes of severe pain that is difficult to control with conventional analgesics, and it is life-threatening due to renal failure and associated cardiovascular and cerebrovascular complications.

With this designation we have made a major achievement, not only for Fabry patients, but also for other pathologies that can benefit from this same approach, made possible by nanotechnology,” explained Nora Ventosa, Scientific Director of NANBIOSIS Unit 6 Biomaterial Processing and Nanostructuring Unit of CIBER-BBN and ICMAB-CSIC who coordinated the project.

Need for new treatments for the disease

This disease, also known as Anderson-Fabry disease, represents the most common lysosomal storage disorder. It is caused by an absence or deficiency of the enzyme α-galactosidase A (GLA), which results in the lysosomal accumulation of globotriaosylceramide (Gb3) and its derivatives in the lysosomes of a wide variety of tissues, responsible for the clinical manifestations. Current treatments consist of intravenous administration of the GLA enzyme, but have limited efficacy and poor biodistribution.

The drug that has been developed is a new nanoformulation of GLA (nanoGLA) that improves efficacy compared to the reference treatment with non-nanoformulated GLA. “The third-generation liposomal product we have developed in the project has demonstrated, at preclinical level, improved efficacy, compared to authorised enzyme replacement treatments, demonstrating that the strategy of supplying the affected cells with the GLA enzyme via the smart nanoliposome is highly successful”, explained Ibane Abasolo, Scientific Coordinator of NANBIOSIS U20 of CIBER-BBN and VHIR, who is responsible for the efficacy studies in the project.

The nanoGLA product was obtained using DELOSTM formulation technology, an innovative platform for the robust production of nanomedicines in an efficient and sustainable manner.

The Committee for Orphan Medicinal Products, the European Medicines Agency’s (EMA) committee responsible for recommending orphan designation of medicines for rare diseases, has considered these results to have a clinically relevant advantage over current enzyme replacement therapies.

The designation of orphan drug, in addition to recognising the significant benefit of the new nanomedicine over products already licensed for Fabry disease, has important implications for the translation of the new therapeutic product from bench to bedside.

Those responsible for these results, including several CIBER-BBN groups, highlight that the new formulation helps to improve treatments, reduce costs, and improve the quality of life of Fabry patients.

Interdisciplinarity and public-private collaboration

The Smart4Fabry project has been running since 2017 thanks to European funding of €5.8 million, from the Horizon 2020 programme. This was possible thanks to the collaboration of several CIBER-BBN groups and NANBIOSIS Units at the Institute of Materials Science of Barcelona (ICMAB-CSIC) with the abouve mentioned NANBIOSIS Unit 6, the Institute for Advanced Chemistry of Catalonia (IQAC-CSIC) with NANBIOSIS Unit 3 of
Synthesis of Peptides Unit
, led by Miriam Royo, the Vall d’Hebron Research Institute (VHIR) with NANBIOSIS Unit 20 and the Institute of Biotechnology and Biomedicine of the Autonomous University of Barcelona (IBB-UAB) with NANBIOSIS Unit 1 Protein Production Platform (PPP), whose work in this project was led by José Luis Corchero. It has also been necessary to contribute knowledge from different academic and business disciplines.

The project consortium also includes public institutions such as the University of Aarhus (Denmark), Technion Israel Institute of Technology (Israel) and Joanneum Research (Austria); and the companies Biokeralty (Spain); Nanomol Technologies SL (Spain); BioNanoNet (Austria), Drug Development and Regulation SL (Spain), the Covance Laboratories LTD group (UK) and Leanbio SL (Spain), which have provided the necessary expertise in nanotechnology and biotechnology, physicochemical characterisation, in vitro and in vivo biological evaluation, formulation and grading of nanomedicines, and pharmaceutical development and production under the guidelines of regulatory agencies.

CIBER and CSIC, promoters of orphan drugs

Orphan Drug Designations (ODDs) seeks to facilitate the arrival of treatments for rare diseases on the market. Several incentives are associated with ODDs, such as market exclusivity, fee reductions and specific scientific advice.

To date, CIBER has promoted eleven orphan drugs designated by the EMA, mainly from the thematic area of Rare Diseases (CIBERER), this being the first from CIBER-BBN.

On the other hand, this is the fourth ODD that the CSIC has obtained, and the first time it refers to a nanoformulated drug.

Orphan drug designation by the European Medicines Agency has several advantages, such as receiving a commercialisation authorisation for 10 years during which similar products cannot be commercialised, the availability of free or low-cost scientific advice and support protocols, and exemption from designation fees. In addition, entities developing orphan drugs have access to specific grants from the European Union and member states’ programmes.

More information

Scientific Culture Unit UCC+i CIBER cultura.cientifica@ciberisciii.es

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NANBIOSIS Scientific Women in the International Day of Women and Girls in Science

Today February 11 is the International Day of Women and Girls in Science, a day to raise awareness of the gender gap in science and technology.

According to the United Nations, while yet women and girls continue to be excluded from participating fully in science, science and gender equality are vital to achieve the internationally agreed development goals, including the 2030 Agenda for Sustainable Development. Thus, in recent years, the international community has made a great effort to inspire and promote the participation of women and girls in science.

NANBIOSIS wants to acknowledge  the efforts made by scientific women who struggle every day to contribute their bit to Science and highlight their essential role in nowadays research. Especially we want to recognize the work of scientists women involved in NANBIOSIS, whatever is the nature of their contribution: technical, scientific development, management, coordination, direction, etc; just to mention some examples:
Neus Ferrer and Mercedes Márquez in the Scientific Direction and Coordination of Unit 1 Protein Production Platform (PPP)
Pilar Marco and Nuria Pascual in the Management and Scientific Coordination of U2 Custom Antibody Service (CAbS) 
Miriam Royo in the Scientific Direction of U3 Synthesis of Peptides Unit
Nora Ventosa and Nathaly Segovia in the Scientific Direction and Technical Coordination of U6 Biomaterial Processing and Nanostructuring Unit
Isabel Oliveira and Teresa Galán in the Coordination of U7 Nanotecnology Unit
Rosa Villa and Gemma Gabriel in the Management and Scientific Coordination of U8 Micro – Nano Technology Unit
Gema Martínez in the Scientific Coordination of U9 Synthesis of Nanoparticles Unit
Fany Peña in the Scientific Coordination of U13 Tissue & Scaffold Characterization Unit
Mª Luisa González Martín and Margarita Hierro in the of Direction and Scientific Coordination of U16 Tissue & Scaffold Characterization Unit
Gemma Pascual and Isabel Trabado in the Coordination of the U17 Confocal Microscopy Service
Isolda Casanova in the Scientific Coordination of U18 Nanotoxicology Unit
Beatriz Moreno in the Scientific Direction of Unit 19 Clinical tests lab
Ibane Abásolo in the Scientific Coordination of Unit 20 In Vivo Experimental Platformt
Verónica Crisóstomo in the Scientific Direction of Unit 24 Medical Imaging 
Ana Paula Candiota in the Scientific Coordination of Unit 25 Biomedical Applications I 
Maria Luisa García in the Scientific Direction of U28 NanoImaging Unit from Bionand, recently incorporated to NANBIOSIS, Anna Aviñó in the Scientific Coordination of U29 Oligonucleotide Synthesis Platform (OSP) – and

Nerea Argarate in the coordination of NANBIOSIS

Thanks to all of you and your teams!

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Smart-4-Fabry final workshop

Next Wedneday, February 3, 2021 will take place the on-line event Smart-4-Fabry Final Workshop.  

Smart-4-Fabry is a european project, coordinated by CIBER-BBN wich has been developed during four years. This project is a sign of cooperation at European level to boost nanomedicine development and translation to clinical stages.

This project is also a clear example of the relevance of access to advanced research infrastructures as NANBIOSIS -ICTS. Four NANBIOSIS units have collaborated and contributed to Smart-4-Fabry development:

“The Fabry disease (FD) is a lysosomal storage disorder (LSD) that currently lacks an effective treatment” as Prof. Nora Ventosa, IP of the project, explained for NANBIOSIS blog – The aim of Smart-4-Fabry is to obtain a new nanoformulation of GLA, that will improve the efficacy and toleration compared to the actual treatment with non-formulated GLA.

In the final workshop experts will talk about how, why and for what the solution proposed by Smart4Fabry was conceived.

Registrations and program at https://smart4fabry.cientifis.com/

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Researchers of NANBIOSIS U3 working in a project to develop molecules with neutralizing properties of SARS-CoV-2

The research group of Chemical Multivalent Systems for Nanomedicine and NANBIOSIS U3 Sinthesys of Peptides Unit, of IQAC-CSIC and CIBER-BBN, participates in a new project that seeks to develop molecules with neutralizing properties of SARS-CoV-2, the cause of the COVID-19 disease, for the treatment of patients requiring hospitalization and clinical supervision due to the severity of the infection.

The project, led by the Public University of Navarra (UPNA) and Navarrabiomed, counts with the participation of researchers from institute of advanced chemistry of catalonia (Institute of Advanced Chemistry of Catalonia), IRB Barcelona (Institute for Biomedical Research), CIBER-BBN (Center for Networked Biomedical Research in the thematic area of Bioengineering, Biomaterials and Nanomedicine) and the IRTA (Institute of Agrifood Research and Technology).

With an expected duration of twelve months, the project brings together a multidisciplinary team: chemical synthesis, protein engineering, structural analysis, cell biology, as well as specialists in BSL3 biosafety conditions (the third level of biosafety for laboratories of a scale of four) to be able to test the potential of these molecules.

These molecules with neutralizing properties of SARS-CoV-2 are based on specific peptides of the ACE-2 receptor capable of reducing or nullifying the infectivity of SARS-CoV-2. The chemical part of the project will be carried out by Miriam Royo and Daniel Pulido (NANBIOSIS Unit 3), with the design and performance of the synthesis of ligands based on the ACE-2 receptor.

The consortium partners hope that “the availability of molecules with high inhibitory efficacy will help substantially to mitigate the socioeconomic impact of the pandemic, due to the persistence and / or future outbreaks of SARS-CoV-2 or other potentially dangerous coronaviruses and routes of similar entry ”, in the words of Jacinto López Sagaseta (Navarrabiomed).

The project has been granted in the “Overcome Covid-19 Fund”. This inicitative was created by Crue Spanish Universities, CSIC and Banco Santander with 8.5 million to bust three strategic axes against covid-19: applied research on the virus and its prevention, social impact and strengthening the technological capacity of the universities, and reducing the digital gap. euros. 35 proposals were have been selected out of the 700 submitted.

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NANBIOSIS Expertise in the IQAC-CSIC Symposium dedicated to the fight against Covid-19 Pandemic

The 4th Annual IQAC Symposium will take place on September 23, 2020. This year will be dedicated to the research projects on COVID-19 that are being carried out in the IQAC-CSIC. It will be a dissemination day aiming to inform the general public about some of the lines of research that are currently being developed in the fight against the COVID-19 pandemic.

The event will be live streamed in Facebook: facebook.com/iqac.csic.es/

Three of the speakers, members of CIBER-BBN NANBIOSIS units at IQAC-CSIC, will explain their work. Pilar Marco and Roger Galve (NANBIOSIS U2 Custom Antibody Service (CAbS) will talk about “Wearable Devices for Detection rapid SARS-CoV-2” and Miriam Royo (NANBIOSIS U3 Synthesis of Peptides Unit shall inform about “Peptide-based chemical tools as diagnostic and therapeutic agents of COVID-19″

Further information and program: http://bit.ly/2ZLVT0o

The Institute for Advanced Chemistry of Catalonia (IQAC) is one of the research centers of the Spanish National Research Council (CSIC). The Institute is located in Barcelona and it was created in 2007 with the mission to perform research of excellence in Chemical Sciences with the broad goal of improving the quality of life. The research developed at IQAC is organized around two main nodes: Biological Chemistry and Nanobiotechnology, many of the investigations carried out by the Research Groups at IQAC lie at the intersection between nodes.

Since 2007 CIBER-BBN and IQAC-CSIC have created four Units of equipment and resources for research wich are part of NANBIOSIS and have been recognized by Spanish Goverment as ICTS (scientific and technical infrastructures, unique in its kind, that are dedicated to high quality research and technological development). The other two units Besides U2 an U3 are: U12 Nanostructured liquid characterization unit, led by Prof. Carlos Rodriguez Abreu and Unir29 Oligonucleotide Synthesis Platform (OSP), led by Prof. Ramón Eritja.

One on the projects in which NANBIOSIS units at CSIC are working in the fight against COVID-19 is CSIC POC4CoV project to develop diagnostic technologies for SARS-COV-2 in which participate 3 of our Units.

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NANBIOSIS researchers featured in the 15th Edition of Spanish Researchers Ranking

The 15th edition of the Webometrics Ranking of World Universities has been published, ranking researchers in Spain as well as Spaniards doing research abroad. A total of 11 Directors of NANBIOSIS units appear on the most recent list, featured on the top 2000. The list is ordered by the h-index, a metric that calculates research impact based on a correlation of papers published and number of citations, and then by number of citations. The result is a list of whose’s publications have had more impact online.

NANBIOSIS researchers featured are Fernando Albericio (#207), scientific director of U3 Synthesis of Peptides Unit, Ramón Martínez Máñez (#342) U26 NMR: Biomedical Applications II, Jaume Veciana (#459) U6 Biomaterial Processing and Nanostructuring Unit, José Luis Pedraz (#906) U10 Drug Formulation unit, Jesús Santamaría (#912) U9 Synthesis of Nanoparticles Unit, Ramón Eritja (#1022) U29 Oligonucleotide Synthesis Platform (OSP), Pablo Laguna (#1153) U27 High Performance Computing, Antoni Villaverde (#1249) U1 Protein Production Platform (PPP), Laura Lechuga (#1511) U4 Biodeposition and Biodetection Unit M.Pilar Marco (#1517), U2 Custom Antibody Service (CAbS), and Josep Samitier (#1836) U7 Nanotechnology Unit.

This list reflects on the impact online publication can have as a tool to share knowledge. 

For further information: here

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Peptide‐Capped Mesoporous Nanoparticles: Toward a more Efficient Internalization of Alendronate.

Osteoporosis is an illness which appears when the osteoblast/osteoclast activities are unbalanced taking place bone resorption (caused by osteoclasts) in higher extension than bone formation (induced by osteoblasts). Alendronate is one of the most used drugs for osteoporosis treatment despite its scarce bioavailability. In an attempt to improve it, gated mesoporous silica nanoparticles, for the controlled release of alendronate, have been synthesized and characterized. These hybrid nanoparticles include labelled alendronate inside the porous, those porous are capped with a peptide designed to be selectively cleaved by cathepsin K enzyme (overexpressed in osteoclasts).

Two CIBER-BBN units of the ICTS NANBIOSIS were implied in the research: the peptide was prepared by U3 Synthesis of Peptides Unit and substances were characterized at U26 NMR: Biomedical Applications II Unit at University of Valencia.

The nanoparticles were internalized by RAW 264.7 macrophages (which could differentiate in osteoclasts) and were able to release its entrapped cargo in the presence of cathepsin K added in the macrophage lysates. From the set with aminopropyl functionalized silica, loaded with nitrobenzofurazan labelled alendronate and capped with the same peptide, 4.2% of the total alendronate amount in contact with the cells is liberated inside them and could produce its therapeutic effect.

Article of reference:

Elena Añón, Ana M. Costero, Pedro Amorós, Jamal El Haskouri, Ramón Martínez‐Mánez, Margarita Parra, Salvador Gil, Pablo Gaviña, M. Carmen Terencio, María Alfonso. Peptide-Capped. Mesoporous Nanoparticles: Toward a more Efficient Internalization of  Alendronate. Chemistry Europe, March 2020

https://doi.org/10.1002/slct.202000417

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A new strategy to synthesize Chol-PEG “on demand”

Researchers of NANBIOSIS U3 Synthesis of Peptides Unit of CIBER-BBN at IQAC-CSIC, led by Miriam Royo, have published an article in the scientific journal ACS Omega that develops a method for the production of cholesterol conjugates to peptide ligands through polyethylene glycol (ND-PEG) not dispersed through a non-hydrolyzable bond.

The developed methodology allows to generate stable, highly pure and well defined Chol-PEG compounds, linked to different lengths of PEG from 4 to 20 units of ethylene oxide through an ether bond. These Chol-PEG compounds were conjugated with different peptides, such as cyclic peptide (RGDfK), gluturation and a Tf1R peptide ligand, preserving the oligomeric purity of the precursors, as demonstrated by analysis by HRMS and NMR. This method allows the systematic synthesis of stable, high-purity Chol-PEGs, bypassing the use of activation groups at each elongation step and thus reducing the number of synthesis steps.

The use of Chol-PEG conjugates has increased in recent years as a component of nanoformulations for applications such as drug administration or imaging systems, due to the ability of Chol to insert into artificial and cell membranes. This ability facilitates the stability of nanovesicles, such as liposomes or others, and entry into cells.

The synthesis of the peptides used in this work was carried out in Unit 3 of NANBIOSIS – ICTS Peptide Synthesis Unit and was developed within the framework of the HORIZON 2020 Smart4Fabry project.

Article of referrence:

Synthesis of Stable Cholesteryl–Polyethylene Glycol–Peptide Conjugates with Non-Disperse Polyethylene Glycol Lengths. Edgar Cristóbal-Lecina, Daniel Pulido, Pau Martin-Malpartida, Maria J. Macias, Fernando Albericio, and Miriam Royo. ACS Omega 2020 5 (10), 5508-5519 https://doi.org/10.1021/acsomega.0c00130

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NANBIOSIS U2, U3 & U29 participate in the POC4CoV project to develop diagnostic technologies for SARS-COV-2

The Spanish Higher Council for Scientific Research (CSIC) will finance the project Point-of-care tests for the rapid detection of SARS-CoV-2 (POC4CoV), whose objective is to have effective diagnostic technologies for Covid-19. The Institute of Microelectronics of Barcelona (IMB-CNM-CSIC), the Institute of Advanced Chemistry of Catalonia (IQAC-CSIC) and the Institute of Materials Science of Aragon (ICMA) participate in it.

The POC4CoV project aims to develop Point-of-Care (POC) devices for the in vitro diagnosis of SARS-COV-2 infection quickly and reliably, thanks to the use of multiplexed systems and the use of particular biomolecular probes. To do this, POC technological platforms will be used in combination with specific capture biomolecules and nanobiotechnological probes (enzyme bioconjugates and biofunctional plasmonic and magnetic nanoparticles), which will allow the simultaneous detection of different biomarkers (viral RNA and antigens, IgM and IgG) related to Covid-19 disease. The biomolecular complexes will be collected at specific points on the devices where the electrochemical or optical signals will be recorded.

The developed POC platforms will undergo analytical and clinical validation in a clinical setting.

Three units of NANBIOSIS (form CIBER-BBN and IQAC-CSIC) will will take an active participation in the project.

NANBIOSIS Unit 2 Custom Antibody Service (CAbS), will produce antibodies against the Spike protein and other virus proteins, trying to maximize the recognition of those epitopes that differentiate SARS-CoV-2 from other Coronaviruses

NANBIOSIS Unit 3 Synthesis of Peptides Unit will synthesize peptidic sequences that will allow to identify towards which epitopes the immune response is directed, which will allow to develop more specific diagnostic methods.

NANBIOSIS Unit 29 Oligonucleotide Synthesis Platform (OSP) has designed probes with oligonucleotide sequences that will allow the capture of viral RNA through the formation of high affinity triplex complexes

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Rare diseases Day February 29: combating Fabry Disease

29 of February is a ‘rare’ date and February, a month with a ‘rare’ number of days, has become a month to raise awareness about rare diseases and their impact on patients’ lives.  Since 2008 thousands of events happen every year all around the world and around the last day of February.

NanoMed Spain Platform and the Hospital of Sant Joan de Déu have organized the NanoRareDiseaseDay to present the latest innovations in the field of Nanomedicine for the treatment and diagnosis of rare diseases (diseases affecting less than 5 people per 10,000 inhabitants). Nora Ventosa, Scientific Director of NANBIOSIS U6 Biomaterial Processing and Nanostructuring Unit  (CIBER-BBN / ICMAB-CSIC) presented Smart4Fabry a European project with the aim of reducing the Fabry disease treatment cost and improve the life-quality of Fabry disease patients

Fabry disease is one of the rare diseases that currently lack a definitive cure. It is cause by lysosomal storage disorders (LSDs):  the deficiency of α-Galactosidase A (GLA) enzyme activity result in the cellular accumulation of neutral glycosphingolipids, leading to widespread vasculopathy with particular detriment to the kidneys, heart and central nervous system.

Smart-4-Fabry has been conceived to obtain a new nanoformulation of GLA, that will improve the efficacy and toleration compared to the actual treatment with non-formulated GLA. Four units of NANBIOSIS participate in the project:

U1 Protein Production Platform (PPP) led by Neus Ferrer and Antony Villaverde at IBB-UAB accomplish the production and purification in different expression systems for R&D purposes.

U3 Synthesis of Peptides Unit led by Miriam Royo at IQAC-CSIC performs all the chemical process of the Smart-4-Fabry  project, i.e. design and synthesis of peptides used as targeting ligands in the nanoliposome formulation

U6 Biomaterial Processing and Nanostructuring Unit led by Nora Ventosa and Jaume Veciana at ICMAB-CSIC undertakes tasks related to the manufacture of the nanoliposome formulation of GLA enzyme and the physico-chemical characterization (this unit counts with plants at different scales, from mL to L, which allow process development by QbD and process scale-up, as well as instrumental techniques for assessment of particle size distribution, particle concentration, particle morphology and stability, and Z-potential)

U20 In Vivo Experimental Platform led by Simó Schwartz and Ibane Abásolo at VHIR to carry out the non-GLP preclinical assays of the project (in vivo efficacy, biodistribution and tolerance/toxicity assays).

For further information about Fabry disease and the Smart4Fabry project: here

Nora Ventosa explaining the progress of the smart4fabry
project on nanoliposomes development for the treatment of Fabry disease
(Pictures by Nanomed Spain)
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