Preclinical development of a nanomedicine candidate for Fabry rare disease treatment to enter clinical phase
Project funded under
The European Innovation Council (EIC)
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
Beneficiary
CSIC
Overall Budget M€
2.5
Lysosomal Storage Disorders - Rare Diseases
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
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
Elisabet González
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
Business model development advice
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)