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News U20

News U20

New equipment adquired to improve NANBIOSIS U20

NANBIOSIS, U20. In Vivo Experimental Platform, led by Dr. Ibane Ibasolo, has been updated and improved as a result of its participation in the project FICTS1420-20, selected by the MICINN for co-financing by the FEDER Program in ICTS 2014-2020 (Equipment for setting standardized immunotoxicology assays for the U20 -NANBIOSIS I17 Action of the Investment Plan-)

A particle particle analyzer equipment (Exoid) has been inslalled at NANBIOSIS U20 for measurement of the concentration and the size of nanoparticles. The Exoid is the latest instrument from the Izon company to measure the physical characteristics of nanoparticles in electrolytic solution. Based on Tunable Resistive Pulse Sensing (TRPS) principles, the equipment is capable of measuring the size of the particles, their concentration and Z potential, through the use of a nanopore that allows measurements to be made in a defined size range.

Finally, a new upright laboratory freezer Liebherr _ SFNe 5227 has been adquired for the storage of the samples and reagents used in the different services of FVPR/U20,

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Workshop on Hyperthermic Oncology

Next Friday, June 9, 2023, a Workshop on Oncological Hyperthermia entitled “Clinical Implementation of Oncological Hyperthermia in Spain and Europe: current situation and prospects” will be held in Barcelona. The location of the event is at the Hotel Front Marítim in Barcelona and the scheduled time is from 9:00 a.m. to 5:30 p.m.

Relevant speakers will present an updated vision on the different modalities of hyperthermia that are being used for cancer treatment. Jorge Contreras (Coordinator of the Spanish Society of Radiation Oncology ) will give a session on the current situation of clinical hyperthermia in Spain. Daniel Ortega (Coordinator of the National Network of Nanotechnology in Translational Hyperthermia) and Teresa Macarulla (Coordinating Investigator of the NoCanTher study on the use of magnetic nanoparticles associated with hyperthermia treatment in pancreatic cancer) will present their experience with a nanotechnological health product in said trial. After a break, Giammaria Fiorentini (Director of the Oncology Unit of the Muraglia di Pesaro Hospital) will give a vision of the use of electro-modulated hyperthermia in pancreatic cancer and in a round table, different professionals will give therir global vision of the application of these techniques from preclinical to clinical implementation.

In addition, regulatory aspects will also be covered. Luc van Hove (Medical, Regulatory and Clinical Affairs expert) will speak from a European perspective on the regulation of medical devices and in vitro diagnostics; while Julia Caro (Head of the area of the National Center for Certification of Sanitary Products) will do so from the perspective of a notified body.

Further information and registration at this link: https://forms.office.com/e/0251iMc2LT

There is additional information at:
-Linkedin https://www.linkedin.com/posts/safenmt_hyperthermia-magnetic-barcelona-activity-7062697943509737472-85wP?utm_source=share&utm_medium=member_desktop.
-Twitter https://twitter.com/abasolo_vhir/status/1659074218850484224

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A discovery in one of the most aggressive cancers will allow more efficient diagnosis

The extracellular vesicles secreted by triple-negative breast cancer stem cells are markers of lung metastasis, according to a study carried out by researchers at CIBER.

The work has been carried out by researchers from various CIBER-BBN groups (Bioengineering, Biomaterials and Nanomedicia), and CIBERONC (CIBER area focused on cancer) has participated in it. The research has been led by Joaquín Seras, from the Vall d’Hebron Research Institute (VHIR), a specialist in targeted drug therapies.

Physicochemical EVs characterization and all the in vivo studies were performed by NANBIOSISunits of CIBER, specifically NTA analysis was carried out at Unit 6 of Biomaterial Processing and Nanostructuring, led by Nora Ventosa at ICMB-CSIC and animal experimentation at Unit 20 “In vivo experimental platform”, led by Ibane Abasolo at VHIR.

The vesicle, in cell biology, is an organelle that forms a small, closed compartment, separated from the cytoplasm by a lipid bilayer just like the cell membrane. The vesicles store, transport or digest cellular products and waste. According to Joaquin Seras, leader of the research: “the identification of this subpopulation of cancerous extracellular vesicles, and their important role in the progression of the disease, will allow in the future to develop systems more effective and less invasive diagnostic methods based on their detection directly from blood samples”.

In different types of tumors, including triple negative breast cancer, it has been observed that the extracellular vesicles generated by tumor cells play an important role in the generation of pre-metastatic niches. Triple negative breast cancer, one of the most aggressive, highly plastic and heterogeneous, is characterized by a significant presence of malignant stem cells.

The study carried out by the Spanish researchers from CIBER with promising results, published in the “International Journal of Cancer”, shows, both in in vitro and in vivo models of the disease, that the vesicles actively contribute to the formation of areas with favorable conditions for the formation of metastases, thus favoring way, the spread of the disease.

Research contributions
In the opinion of Joaquin Seras, the great contribution of this work is that it “describes how the extracellular vesicles secreted by certain subpopulations of cancer cells, specifically those derived from cancer stem cells, have the potential to modify the microenvironment of the future metastatic niche to promote tumor growth.

In other words, continues the leader of the study: “the research sheds new information on the pathogenic mechanism of the disease, and suggests these extracellular vesicles as markers with diagnostic potential. It should be noted that these nanoparticles are secreted into the bloodstream by tumor cells, and effective capture and identification would allow them to be exploited as a diagnostic tool”.

On the characterization of extracellular vesicles of cancer cells
The complex composition and functional differentiation of cancer cells in a tumor also increases the heterogeneity of the subsets of vesicles secreted by cancer.

This phenomenon is particularly relevant in triple negative breast cancer, one of the most aggressive, highly plastic and heterogeneous cancers, characterized by a significant presence of malignant stem cells. However, until now the diversity of the vesicles secreted by cancer cells had not been studied, a diversity that is closely related, in turn and as the study shows, to cellular heterogeneity in triple-negative tumors.

The importance of the CIBER study lies at this point: the vesicles secreted by different tumor subpopulations and grouped by their degree of differentiation show fundamentally different activities in terms of their impact on cancer progression.

In the investigation, the extracellular vesicles secreted by up to three different types of neoplastic cells have been isolated and characterized, observing different bioburdens for each type, with the consequent differential effect on stromal cells. In addition, and as the study shows, cancer stem cell-derived vesicles contribute to converting healthy lung cells into receptive niches for the metastatic growth of cancerous breast cells.

Article reference:

González-Callejo P, Gener P, Díaz-Riascos ZV, Conti S, Cámara-Sánchez P, Riera R, Mancilla S, García-Gabilondo M, Peg V, Arango D, Rosell A, Labernadie A, Trepat X, Albertazzi L, Schwartz S Jr, Seras-Franzoso J, Abasolo I. Extracellular vesicles secreted by triple-negative breast cancer stem cells trigger premetastatic niche remodeling and metastatic growth in the lungs. Int J Cancer. 2023 Jan 27. doi: 10.1002/ijc.34447. Epub ahead of print. PMID: 36705298.

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Rare diseases international day 2023: some NANBIOSIS contributions

Today is the international day of rare diseases, a day to raise awareness and instigate change for people living with a rare disease. From NANBIOSIS we want to sume to this celebration and higtligh our commitment to helping people with rare diseases through research.

Dr. Ibane Abasolo, Scientific Director of NANBIOSIS U20, was at the WORLDSymposia conference last week in Orlando (FL, USA), where the latest advances in preclinical study and clinic of lysosomal storage diseases were explained. There, she presented the results obtained in the Smart4Fabry project coordinated by the CIBER-BBN where nanoliposomes were developed for the treatment of Fabry disease. The work, entitled “Preclinical Validation of Nanoliposomes for ERT for Fabry disease”, was a result of the collaboration of the groups of Dr. Ventosa and Dr. Corchero, both from CIBER-BBN, and the participation of units U1, U3, U6, and U20 of the ICTS Nanbiosis.

In addition, today Dr. Abasolo participated in the Nano Rare Day session, organized by the NanoMedSpain platform and the Barcelona Bioengineering Institute (IBEC) at the Sant Joan de Deu Hospital in Barcelona, presented the work entitled “Use of natural and artificial nanoparticles for the treatment of lysosomal storage diseases”, where in addition to nanoliposomes, she also detailed how extracellular vesicles can be a good vehicle to improve replacement enzyme therapy in lysosomal diseases.

Also Dr. Juan Pablo Salvador from NANBIOSIS U2 CAbS has presented at in the Nano Rare Day session his talk on “Quorum Sensing to improve the management of cysticfibrosis“, explaining the difficulty of quickly identifying bacterial infections, which are common in patients with Cystic Fibrosis. In this sense, “Quorum Sensing”, a microbial communication mechanism through which the cells themselves regulate the expression of genes based on cell density, can help identify biomarkers and improve the management of cystic fibrosis.

Related news: Fabry Desease in the Rare Disease Day: A New Hope

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1st Nanomedicine Forum of CIBER-BBN/NANBIOSIS and CSIC Nanomed Conection

During the days 30 of June and 1st of July took place in Barcelona, in the auditorium of the Institute of Advanced Chemistry of Catalonia (IQAC-CSIC), the 1st Forum on Nanomedicine gathering scientists from the CSIC net Nanomed Conection and from the CIBER-BBN and its ICTS NANBIOSIS.

This forum brought toguether researchers from the most eminent national research centers in nanomedicine, that during the two days meeting presented their works and findings and discussed the impact of nanomedicine in the fields of drug delivery, diagnosis and therapy.

The workshop was open by the Director of IQAC-CSIC,  Jesús Joglar, the  Scientific Coordinator of Nanomed Conection, Fernando Herranz, and the Scientific Director of CIBER-BBN, Ramón Martínez Máñez.

18 research groups gave their talks distributed in four sessions:

  • Nanobiotechnological solutions for diagnosis and therapy
  • Drug delivery nanosystems
  • Applications for oncology 
  • Nanomedicine & other frontier applications

The presentations aroused great interest and futher debate among the attendees present in the auditorium (around 50) and the on line participants (The event was also broadcast online previous registration with more than 125 registrations received).

The videos of the presentations will be soon available in the NANBIOSIS youtube channel.

Here we highlight the eight talks by researchers from NANBIOSIS units:

The first session of Nanobiotechnological solutions for diagnosis and therapy,  started  with the talk by Montserrat Rodríguez from Nb4D group NANBIOSIS U2 CAbS, from CIBER-BBN and IQAC-CSIC, entitled “Targeting aromatic amino acid metabolism for the early diagnosis of neurological diseases”, presenting their results on in vitro samples, on thermal power characterization experiments to study the thermal efficiency of non-sinusoidal stimulation and on efficiency characterization experiments in cell cultures with cancer cell liness.

Also in this session chaired by Miriam Royo, Scientific Coordinator of NANBIOSIS U3 Synthesis of Peptides Unit of  CIBER-BBN and IQAC-CSIC,  took place an interesting and passionate talk by Ramón Eritja, Scientific Director of NANBIOSIS U29 Oligonucleotide Synthesis Platform (OSP)

In the last years, interest in therapeutic applications of oligonucleotides has increased enormously, especially after the development of messenger RNA vaccines in response to the COVID-19 pandemic. In this way, metabolic diseases such as dyslipidemia and hereditary diseases such as Duchenne muscular dystrophy have been successfully addressed. The NANBIOSIS  Oligonucleotide Synthesis Platform (OSP) focuses on the design, synthesis and characterization of modified oligonucleotides, in order to enhance the therapeutic properties of the oligonucleotides and to improve the control of gene expression. Ramon Eritja presented their most recent results in the development of new conjugates with antiproliferative activity and in the design of DNA probes for the detection of viral genomes.


In the session of “Nanomedicine and other frontiers applications”, chaired by María del Puerto Morales Herrero (ICMM-CSIC), Elena Martínez Fraiz,  from the Nanobioengineering group of CIBER-BBN and IBEC running NANBIOSIS Unit 7 of Nanotechnology, presented  a nanostructured surface able to produce multivalent effects of surface-bound ephrinB1 ligands on the dynamics of oligomerization of EphB2 receptors  whic can benefit applications such as the design of new bioactive materials and drug-delivery systems.

The session of Drug delivery nanosystems, chaired by Ramón Martínez Máñez, began with the talk by Vanessa Díaz Riascos, presesnting the in vivo efficacy, biodistribution and toxicity testing of nanomedicines at NANBIOSIS U20 FVPR, of CIBER-BBN and VHIR, explaining how their texting expertise and their in vivo and ex vivo fluorescence imaging techniques facilitate a rapid and efficient preclinical development of candidates, reducing considerably the time and costs of conventional developments.

Santiago Grijalvo Torrijo, from NANBIOSIS U12 Nanostructured liquid characterization unit expoke about Nano-emulsion-derived polymeric carriers for biomedical applications also discussing the impact of the protein corona on colloidal stability, antioxidant activities, cytotoxicity and cellular uptake of drug-loaded nanoparticles.

Antoni Llopis Lorente, (NANBIOSIS U26 NMR: Biomedical Applications II), expoke about Gated silica nanoparticles for controlled release. Chemical communication, based on the exchange of molecules as messengers, allows different entities to share information, cooperate and orchestrate collective behaviors. Communication using chemical messengers (such as neurotransmitters, hormones and pheromones) is the main way of communication across the natural world; yet engineering chemical communication between micro/nanosystems is a key emergent topic in micro/nanotechnology, biomimicry and related areas. Santiago explainined recent progress by their group in the development of engineered particles for chemical communication and nanomedicine applications.

And closing the session, Mariana Köber (Nanomol Group –NANBIOSIS U6 of Biomaterial Processing and Nanostructuring Unit  from CIBER-BBN and ICMAB-CSIC) gave a talk on Quatsomes as versatile nanovesicles for biomedical applications.

In the session of Applications for Oncology, Pilar Martín Duque from NFP group – NANBIOSIS U9 Synthesis of Nanoparticles Unit of CIBER-BBN and INMA-CSIC, gave a very interesting talk explained their approach and recent progress on the search of trojan horses for an improved theragnosis of cancer.

Here we want to thank the Institute of Advanced Chemistry of Catalonia (IQAC-CSIC) for hosting this event and for the help in its preparation and development.

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Targeting of the breast cancer stem cells to improve the treatment of triple negative breast cancer

On April 1st PhD candidate Patricia Cámara Sánchez defended her doctoral thesis entitled “Targeting of the breast cancer stem cells to improve the treatment of triple negative breast cancer“, where ICTS-Nanbiosis Unit 20 participated in the in vivo assays. The thesis was supervised by Dr. Ibane Abasolo  (scientific director of Unit 20 from CIBER-BBN and VHIR). Nanbiosis was also present within the jury, with Dr. Ana Paula Candiota (scientific coordinator of Unit 25 from CIBER-BBN and UAB) acting as secretary.

Patricia Cámara graduated as biochemist, did the master’s degree in Translational Biomedical Research from VHIR-UAB. Shortly after, started the PhD, which was aimed at improving the treatment of very deadly subtype of breast cancer by using different nanoformulations to specifically target the cancer stem cells. The now doctor Cámara-Sánchez screened up to 20 small drugs with anti-cancer stem cell activity, found synergistic ratios with conventional chemotherapeutic agents, and finally developed polymeric micelles encapsulating selected drugs. During the discussion of the dissertation, the need of additional in vivo assays was highlighted, as well as the potential use of MR provided by the U25, to explore non-invasively the metabolomic differences between cancer stem cells and regular cancer cells.

‘I’m very grateful for the opportunity to have been part of this amazing project. It has been a very enriching experience’, she explains. ‘After 5 years of research, I finish the PhD feeling very proud of having contributed to the fight against this aggressive subtype of breast cancer’, she adds. From now on, new research lines will bring forward the synergies between both units of NANBIOSIS, reinforcing a collaboration started several years ago and reflected in joint papers.

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New equipment installed in NANBIOSIS U20

NANBIOSIS, U20. In Vivo Experimental Platform, led by Dr. Ibane Ibasolo, has been updated and improved as a result of its participation in the project FICTS1420-20, selected by the MICINN for co-financing by the FEDER Program in ICTS 2014-2020 (Equipment for setting standardized immunotoxicology assays for the U20 -NANBIOSIS I17 Action of the Investment Plan-)

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The efficacy of nanoparticles combined with chemotherapy against pancreatic cancer

Pancreatic ductal adenocarcinoma is a highly devastating orphan disease with poor prognosis even when diagnosed early (The survival rate after 5 years (< 5%) has not changed over the last 30 years, despite tremendous research efforts) The european project NoCanTher, in wich participates NANBIOSIS U20 of (VHIR and CIBER-BBN) is a nanotechnology-based approach aimed to scale-up under GMP conditions and evaluate a nanoformulation for a combined therapy with chemotherapy against this pancreatic cancer.

The international clinical study, carried out in the framework of the project is now incorporating the first pacient in Spain with this type of nanotherapy.

The clinical study is based on the results obtained in the preclinical phase of the NoCanTher project, with the group of the CIBBIM (of CIBER-BBN and VHIR, led by Dr. Ibane Abasolo). This new technology based on magnetic nanoparticles increases the accessibility of chemotherapy to pancreatic tumors. Researchers have developed magnetic iron nanoparticles that, under an alternating magnetic field, generate heat – magnetic hyperthermia. This heat is able to be used because the tumor cells are still more sensitive to the standard treatment with chemotherapy and, therefore, in greater efficiency, and ultimately it is possible to directly destroy these cells.

As explained by Dra. Ibane Abasolo, Scientific Director of Nanbiosis unit 20 of CIBER-BBN and VHIR: NANBIOSIS has played an important role in this project in relation with the preclinical in vivo trials in animal models (mice). We made human pancreatic cancer cells grow and studied the efficacy of nanoparticles and their combination with chemotherapy. Specifically, we saw that i) tumor heating by magnetic hyperthermia slowed down tumor growth, but that it was necessary to combine this treatment with conventional chemotherapy to have a better effect, that ii) the hyperthermia-chemotherapy sequence that worked best (it is better to warm up first and treat with chemotherapy later than to put chemotherapy first) and iii) that the combination of hyperthermia/chemotherapy was beneficial because it greatly reduces the stroma of the tumor and makes it easier for chemotherapy to reach tumor.

The NoCanTher project is coordinated by IMDEA Nanociencia (Madrid) and includes the participation of eleven national and international centers: BioKeralty Research Institute (Miñano), ImmuPharma (London), Chemicell (Berlin), University Hospital (Jena, Germany), Resonant Circuits (London), Vall d’Hebron Research Institute (VHIR) (Barcelona), Vall d’Hebron Institut d’Oncology (VHIO) (Barcelona), Trinity College (Dublin), Paris Diderot University (Paris), Hospital Universitari of Fuenlabrada (Madrid). The initiative is funded by Horizon 2020 (GA:685795).

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Open Call Focus on Innovative MedTech using Nanotechnology

Start-ups / SMEs! Are you working on innovative medical products using nanotechnology? This is your call for development and marketing solutions!

Last week took place the SAFE-N-MEDTECH Open Call on line info session with the idea to present the open call with a bit more detail. A few presentations and specially a time for questions and answers were organized by Anaïs Le Corber, Network Manager of CEBERG, the council of European Bioregions, partner of the project.

The webinar was recorded and it is available here:

First of all, Angel del Pozo, Coordinator of the SAFE-N-MEDTECH, gave an insight of the project.

Then, Miguel Duarte from IMM (Portugal), the business development partner of the project, presented the services offered

After him, Qwentin Pankhurst, chairman of Resonant Circuits Ltd. explained his experience as user, having a tase case in the project.

Finaly, before the space for questions and answers, Ibane Abasolo, Scientific Director of NANBIOSIS U20, from CIBER-BBN and Vall D’Hebron Institute Research in Barcelona, presented with more detail the clinical validation services available in this open call.

SAFE-N-MEDTECH OITB  Safety testing in the life cycle of nanotechnology-enabled medical technologies for health is a H2020 project with the objective of providing services and support to companies and other organizations for accelerating the development and commercialization of innovative MedTech solutions based on nano-enabled technology.

How can this call suit you?

Qwentin Pankhurst explained his experience: “As a medtech start-up we do not have the external experience to cover all the safety related aspects of product development for MDR compliance, we needed clear uncomplicated advance and solutions, ideally we needed a “one-stop-shop”…

“Even if a client accesses only a part of the offering, the fact that the OITB is seeking to cover the entire pathway from design input to clinical testing is important as it underpins a connected-thinking approach.”

Submission deadline: March 21st 2022, 23:59 CET

The time line: It is espected that the evaluation process will take around one month. Then NDAs will be signed before starting the work, which could be carried during one year approximatly.

The technology assesment area of the SAFE-N-MEDTECH OITB well carry out a first examitanion toguether with the applicant of the available data, the quality of the data, the needs of the product an other relevant aspects. A technology assitant proposal will ve developed and the services needed will be defined toguether with the client.

Call documents:

  • Guidelines for Applicants The Guide for Applicants contains the basic information needed to guide you in preparing a proposalfor submission to the SAFE-N-MEDTECH Open Call. It gives an introduction on how to structureyour proposal. It also describes how to submit the proposal and the evaluation criteria.
  • Application Form

This call uses funds obtained within the scope of the SAFE-N-MEDTECH project funded by European Union´s Horizon 2020 Research and Innovation Program. (Grant Agreement No. 814607)

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Fabry Desease in the Rare Disease Day: A New Hope


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 with the aim of improving equity and reducing stigmatization for people who live with more than 6,000 rare diseases.


Rare diseases are pathologies or disorders that affect a small part of the population (less than 5 per 10,000 inhabitants) and generally have a genetic component. They are also known as orphan diseases.

Diseases present a series of particular symptoms, and it is very difficult to diagnose what their true cause is. These disorders or alterations that patients present must be evaluated by a specialist, depending on each case.

Today 5% of the world population suffer from them. This translated into numbers, corresponds to approximately 300 million affected.

A patient with a rare disease waits an average of 4 years to obtain a diagnosis, in 20% of cases it takes 10 or more years to achieve the proper diagnosis.


To combat this disease, patients need to be treated with so-called orphan drugs. They serve to prevent and treat pathology. Its composition is based on biotechnological compounds whose manufacture is very expensive and not profitable for companies. For this reason, cooperation of governments is needed as well as financial incentives to encourage pharmaceutical companies to develop and market medicines to make these treatments accessible to a greater number of people.


Fabry is one of the rare diseases that currently lack a definitive cure. Symptoms may include episodes of pain, especially in the hands and feet (acroparesthesias); small dark red spots on the skin called angiokeratomas; decreased secretion of sweat (hypohidrosis); opacity of the cornea (cataracts) and hearing loss. Internal organs such as the kidney, heart, or brain may be involved, resulting in progressive kidney damage, heart attacks, and strokes.

Fabry disease is a lysosomal storage disease arising from a deficiency of the enzyme α-galactosidase A (GLA). The enzyme deficiency results in an accumulation of glycolipids, which over time, leads to cardiovascular, cerebrovascular, and renal disease, ultimately leading to death in the fourth or fifth decade of life. Currently, lysosomal storage disorders are treated by enzyme replacement therapy (ERT) through the direct administration of the missing enzyme to the patients.


CIBER-BBN, through the researcher Nora Ventosa has coordinated the european project “Smart-4-Fabry” developed during 2017-2021, the proyect was undertaken by a consortium formed by ten partners, including private companies and public institutions in Europe and Israel, with a Horizon 2020 financial programme by the European Commission (H2020-NMBP-2016-2017; call for nanotechnologies, advanced materials, biotechnology and production; Proposal number: 720942-2).

In view of their advantages as drug delivery systems, liposomes are increasingly being researched and utilized in the pharmaceutical, food and cosmetic industries, but one of the main barriers to market is their scalability.

Depressurization of an Expanded Liquid Organic Solution into aqueous solution (DELOS-susp) is a compressed fluid-based method that allows the reproducible and scalable production of nanovesicular systems with remarkable physicochemical characteristics, in terms of homogeneity, morphology, and particle size. The objective of this work was to optimize and reach a suitable formulation for in vivo preclinical studies by implementing a Quality by Design (QbD) approach, a methodology recommended by the FDA and the EMA to develop robust drug manufacturing and control methods, to the preparation of α-galactosidase-loaded nanoliposomes (nanoGLA) for the treatment of Fabry disease.

Through a risk analysis and a Design of Experiments (DoE), researechers obtained the Design Space in which GLA concentration and lipid concentration were found as critical parameters for achieving a stable nanoformulation. This Design Space allowed the optimization of the process to produce a nanoformulation suitable for in vivo preclinical testing.

The new nanoformulation developed by Smart4Fabry for the treatment of Fabry disease achieved the ODD (Orphan Drug Designation) by the European Commission. The new nanomedicine is more effective and has a better biodistribution than the current treatments, based on enzyme replacement. The new nanomedicine is based on a nanovesicle that protects the enzyme and achieves a better cell internalisation, thus reducing the doses needed, the total cost and improving the quality of patients.

Four units of NANBIOSIS participated in the project:

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

– U3 Synthesis of Peptides Unit led by Miriam Royo at IQAC-CSIC performed 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 at ICMAB-CSIC developed 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 Ibane Abásolo at VHIR carried out the non-GLP preclinical assays of the project (in vivo efficacy, biodistribution and tolerance/toxicity assays).


Researchers of CIBER-BBN and NANBIOSIS, led by Nora Ventosa, are currently participating in another european project, PHOENIX “Enabling Nano-pharmaceutical Innovative Products” in the framework of which this novel nanomedicine developed under the Smar4Fabry project and designed as Orphan Drug by the EMA, will be scaled-up and manufactured under GMP to enable its clinical testing.

Articles of reference:

Josep Merlo-Mas, Judit Tomsen-Melero, José-Luis Corchero, Elisabet González-Mira, Albert Font, Jannik N. Pedersen, Natalia García-Aranda, Edgar Cristóbal-Lecina, Marta Alcaina-Hernando, Rosa Mendoza, Elena Garcia-Fruitós, Teresa Lizarraga, Susanne Resch, Christa Schimpel, Andreas Falk, Daniel Pulido, Miriam Royo, Simó Schwartz, Ibane Abasolo, Jan Skov Pedersen, Dganit Danino, Andreu Soldevila, Jaume Veciana, Santi Sala, Nora Ventosa, Alba Córdoba, “Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method”, The Journal of Supercritical Fluids, Volume 173, 2021, 105204, https://doi.org/10.1016/j.supflu.2021.105204.

Judit Tomsen-Melero, Solène Passemard, Natalia García-Aranda, Zamira Vanessa Díaz-Riascos, Ramon González-Rioja, Jannik Nedergaard Pedersen, Jeppe Lyngsø, Josep Merlo-Mas, Edgar Cristóbal-Lecina, José Luis Corchero, Daniel Pulido, Patricia Cámara-Sánchez, Irina Portnaya, Inbal Ionita, Simó Schwartz, Jaume Veciana, Santi Sala, Miriam Royo, Alba Córdoba, Dganit Danino, Jan Skov Pedersen, Elisabet González-Mira, Ibane Abasolo, and Nora Ventosa. Impact of Chemical Composition on the Nanostructure and Biological Activity of α-Galactosidase-Loaded Nanovesicles for Fabry Disease Treatment, ACS Appl. Mater. Interfaces 2021, 13, 7, 7825–7838 ( https://doi.org/10.1021/acsami.0c16871).

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