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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

Beneficiary

CSIC

INSTITUTE OF MATERIALS SCIENCE OF BARCELONA (ICMAB)

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

Team

Nora Ventosa

Nora Ventosa

Project coordination, management, and spin-off creation
in
Elisabet González

Elisabet González

Head of Preclinical development, FIH Clinical trial Design, and Regulatory/ethics aspects
in
Enrique Vilalta

Enrique Vilalta

Business Developer
in
Judit Morlà

Judit Morlà

Scientific Project Manager
in
Juidt Tomsen

Judit Tomsen

Nanoformulation and manufacturing responsible
in
Aida Carreño

Aida Carreño

Analytical development responsible
in
Guillem Vargas

Guillem Vargas

Financial management and communication/dissemination
in
Guillem García

Guillem García

Pharmaceutical development support
in
Angélica Pichardo

Angélica P. Pichardo

Analytical development support
in
Alfonso del Rey

Alfonso del Rey

Contact with CSIC headquarters
in
Anna May

Anna May

Communication/dissemination responsible
in

Collaborative partnership

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 coordinator

Leanbio

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

Programa Indústria del Coneixement

Generalitat de Catalunya

Business model development advice

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

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

Programme: HORIZON.3.1 - The European Innovation Council (EIC)