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Preclinical development of a nanomedicine candidate for Fabry rare disease treatment to enter clinical phase

Project funded under
The European Innovation Council (EIC)

NanoGLA

NanoGLA is a patented protected nanopharmaceutical product for the treatment of Fabry disease. It was designated as Orphan Medicinal Product by the EMA in 2021.

Platform

The liposomal platform offers high versatility to expand its applicability to other rare diseases, giving competitive advantages to yield novel therapies.

From bench to bedside

Bring the nanoGLA towards the market and the commercialisation of the patent protected nanoencapsulation platform to generate new product candidates for the treatment of rare diseases.

Months

24

PERIOD: NOV 2023 - OCT 2025

Overall Budget M€

2.5

EIC TRANSITION

Lysosomal Storage Disorders - Rare Diseases

Lysosmal Storage Diseases (LSD) are characterized by a lysosomal disfunction
LSDs are all rare diseases (RD), but together they represent a serious global health problem
There are multiple LSD, characterized by the deficiency of different enzymes in the lysosome
Fabry
Pompe
Gaucher
Hunter
Sanfilippo

Fabry Disease

Cause

Fabry disease is caused by a defficiency of the GLA (α-galactosidase-A) protein/enzyme

Current treatment

Enzyme replacement as main therapy of Fabry disease, but:

  • low efficiency
  • not accessible to brain
  • need of frequent dosing
  • poor biodistribution profile

Symptoms and nanoGLA treatment

Smart4Fabry EU funded project was the previous project devoted to design the nanoGLA l treatment for the Fabry disease. Herein, a video, prepared during this project, to disseminate the Fabry disease.

Project

Benefits of our nanoliposomal encapsulation platform
Our Solution for Fabry disease: nanoGLA

Work Plan

In total, six work packages (WPs) will work together to ensure the success of the project during the two years that the Nano4Rare project will be active

Preclinical development 

This WP will validate the methods used in the project and the toxicological profile of nanoGLA.

First in human clinical trial design

A specific study plan for the first phase 1 clinical trials must be prepared

Regulatory aspects and ethics

The regulatory and ethical aspects of the first-in-human clinical trials must be addressed in this WP

Business development

A business plan will be prepared for the spin-off that must be incorporated

Dissemination & communication

The Nano4Rare project must be made visible and its results disseminated to the targeted stakeholders.

Project and data management

Resources need to be optimised in order to link them with the other WPs and the European Comission

Nano4Rare Partners

The Institute of Materials Science of Barcelona (ICMAB-CSIC) is a Spanish research center of the Spanish National Research Council (CSIC) specialized in the development of advanced materials for a wide range of applications. Within the institute, the Nanomol-bio group focuses on designing and developing organic materials for biomedical applications, such as nanosystems for drug delivery and release.

Affiliated Entity of CSIC

Delbios Pharmaceuticals focuses on the research, development, production, and marketing of technological solutions in the field of nanomedicine for the treatment of rare diseases.

People

Elisabet González

Elisabet González

PI CISC and Project co-coordinator - CSIC
in
Nora Ventosa

Nora Ventosa

CEO-CSO, PI DELBIOS and Project co-coordinator - DELBIOS
in
Enrique Vilalta

Enrique Vilalta

Business Developer - CSIC
in
Judit Morlà

Judit Morlà

Scientific Project Manager - CSIC
in
Juidt Tomsen

Judit Tomsen

Nanoformulation and Manufacturing Lead - CSIC
in
Aida Carreño

Aida Carreño

Analytical Development Lead - CSIC
in
Guillem Vargas

Guillem Vargas

Project Assistant - CSIC
in
Guillem García

Angela Cortese

Pharmaceutical Development Assistant - CSIC
in
Angélica Pichardo

Angélica P. Pichardo

Analytical Development Support Analyst - CSIC
in
Alfonso del Rey

Alfonso del Rey

CSIC Liaison Officer - CSIC
in
Anna May

Anna May

Communication and Dissemination Manager - CSIC
in

Collaborators and services suppliers

Nanomol Technologies

Industrial nanoencapsulation manufacturing. Business partner, and EU Phoenix OITB Partner.

Vall d'Hebron

Non-GLP preclinical testing (in vitro and in vivo) and clinial advice on pharmacologic testing.

MyBiotech

CDMO that offers clean room facilities for nanomedicine production and EU Phoenix OITB co-coordinator.

Leanbio

CDMO for the development and manufacturing of therapeutic enzymes and EU Phoenix OITB partner.

Unilabs York Bioanalytical Solutions

Specialists in developing analytical methods for detecting biomolecules.

SPARTA Biodiscovery

A spin-out company that uses SPARTA® technology to characterise the addition of molecules to formulations.

KYMOS group

Photoirradiation studies and bioanalytical services of nanoformulations.

BioNanoNet

A non-profit research organisation that provides toxicological and regulatory advice. EU Phoenix OITB partner.

Luxembourg Institute of Science and Technology

Analysis of biomarkers of the Fabry disease. EU Phoenix OITB coordinator.

Creatio

CDMO specialised in developing advanced therapies.

Medical Associations of LSD or RS

Linked projects

EU-funded project:
PHOENIX-OITB (953110)
March 2021 - Feb 2025

EU-funded project:
Smart4Fabry (720942)
Jan 2017 - Dec 2020

AGAUR-Cat-funded project:
Innova4Fabry (2021 INNOV 00053)
Nov 2022 - March 2024

Related publications

Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease

Authors: Judit Tomsen-Melero, Marc Moltó-Abad, Josep Merlo-Mas, Zamira V. Díaz-Riascos, Edgar Cristóbal-Lecina, Andreu Soldevila, Thomas Altendorfer-Kroath, Dganit Danino, Inbal Ionita, Jan Skov Pedersen, Lyndsey Snelling, Hazel Clay, Aida Carreño, José L. Corchero, Daniel Pulido, Josefina Casas, Jaume Veciana, Simó Schwartz Jr., Santi Sala, Albert Font, Thomas Birngruber, Miriam Royo, Alba Córdoba, Nora Ventosa, Ibane Abasolo, and Elisabet González-Mira

Journal: Science Advances (2024), 10:50

DOI: 10.1126/sciadv.adq4738

Liposomal formulations for treating lysosomal storage disorders

Authors: Judit Tomsen-Melero, Josep Merlo-Mas, Aida Carreño, Santi Sala, Alba Córdoba, Jaume Veciana, Elisabet González-Mira, and Nora Ventosa

Journal: Advanced Drug Delivery Reviews 190 (2022) 114531

DOI: 10.1016/j.addr.2022.114531

Impact of Chemical Composition on the Nanostructure and Biological Activity of α-Galactosidase-Loaded Nanovesicles for Fabry Disease Treatment

Authors: 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 Jr., Jaume Veciana, Santi Sala, Miriam Royo, Alba Córdoba, Dganit Danino, Jan Skov Pedersen, Elisabet González-Mira, Ibane Abasolo, and Nora Ventosa

Journal: ACS Appl. Mater. Interfaces (2021) 13, 7825−7838

DOI: 10.1021/acsami.0c16871

Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method

Authors: 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 Jr., Ibane Abasolo, Jan Skov Pedersen, Dganit Danino, Andreu Soldevila, Jaume Veciana, Santi Sala, Nora Ventosa, and Alba Córdoba 

Journal: The Journal of Supercritical Fluids (2021) 173,105204

DOI: 10.1016/j.supflu.2021.105204

α-Galactosidase-A Loaded-Nanoliposomes with Enhanced Enzymatic Activity and Intracellular Penetration

Authors: Ingrid Cabrera, Ibane Abasolo, José L. Corchero, Elisa Elizondo, Pilar Rivera Gil, Evelyn Moreno, Jordi Faraudo, Santi Sala, Dolores Bueno, Elisabet González-Mira, Merche Rivas, Marta Melgarejo, Daniel Pulido, Fernando Albericio, Miriam Royo, Antonio Villaverde, Maria F. García-Parajo, Simó Schwartz Jr., Nora Ventosa, and Jaume Veciana

Journal: Adv. Healthcare Mater. (2016), 5, 829–840

DOI: 10.1002/adhm.201500746

Contact

Institut de Ciència de Materials de Barcelona (ICMAB-CSIC)

  • Carrer dels Til·lers s/n, Campus de la UAB, 08193 Bellaterra, Barcelona, Spain

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This project has received funding from the European Union’s Horizon Europe research and innovation programme under grant agreement n°101136772.

Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or the European Research Executive Agency. Neither the European Union nor the European Research Executive Agency can be held responsible for them.