HEALTH is partner in H2020 project – SMART4FABRY – smart functional GLA nanoformulation for Fabry disease
Smart4Fabry aims to develop a new treatment for the Fabry disease which belongs to the group of so-called lysosomal storage disorders, a group of rare diseases that currently lack a definitive cure. In the case of Fabry disease, the deficiency of α-galactosidase A (GLA) enzyme activity results in an impairment of the kidneys, the heart and the nervous system. The current treatment for Fabry disease is an enzyme replacement therapy, in which patients depend on genetically engineered GLA which has a low efficacy in the brain when applied exogenously through intravenous infusions. Smart4Fabry aims to develop a new, liposomal nanoformulation of GLA, a nano-GLA, that will increase effective delivery of GLA across biological membranes.
As a partner in the consortium, the institute HEALTH will perform comparative studies testing different nanoformulations by using its patented cerebral open-flow microperfusion (cOFM) method. The main focus of these studies is to test the efficiency of different nanoformulations when crossing the blood-brain barrier.
- Project duration: 48 months
- Project start: January 2017
- Coordinator: CIBER-BNN (Spain)
- Partners:JOANNEUM RESEARCH (Austria), Aarhus University (Denmark), BIOPRAXIS Research (Spain), BioNanoNet (Austria), DDR (Spain), Technion-Israel Institute of Technology (Israel),
Covance (UK), LeanBioPro (Spain), NANOMOL TECHNOLOGIES (Spain)