Smart4Fabry
PROJECT DURATION:
09/2017
—
03/2024
TOTAL PROJECT DURATION:
48 Monate
The Project
The aim of the Smart4Fabry project was to develop a new therapy for Fabry disease, which belongs to the group of so-called lysosomal storage diseases, a group of rare diseases for which there is currently no definitive cure.
Our Role in the Project
As a partner in the consortium, HEALTH has compared various nanoformulations in studies in which the patented cerebral open microperfusion (cOFM) was used. The main focus of these studies was to test the effectiveness of different nanoformulations during transport across the blood-brain barrier.
Research groups
Downloads
Customer/Funding Organization
European Commission
Project Partner
12 European Partner
Project Details
In Fabry disease, the lack of α-galactosidase A (GLA) enzyme activity leads to impairment of the kidneys, heart and nervous system. The current treatment for Fabry disease is enzyme replacement therapy, in which patients rely on genetically modified GLA, which has low efficacy in the brain when administered exogenously by intravenous infusion. Smart4Fabry is working on the development of a new liposomal nanoformulation of GLA, a nano-GLA that increases the effective transport of GLA across the blood-brain barrier.
Learn more: Smart multifunctional GLA-nanoformulation for Fabry disease
