The project has started on January 1st, 2017, and will run for 4 years with 10 partners and a budget of 5,800,000 €.
The research of Smart-4-Fabry project concerns development of drug delivery systems based on liposomes for treatment of Fabry disease. Advanced formulation and characterization techniques will be used for achieving excellent quality control over the assembly of the different molecular components of a new liposomal nano-formulation which contains the α-Galactosidase A (GLA) enzyme as the active component. Fabry disease belongs to a group of lysosomal storage disorder (LSD) diseases that currently lack a definitive cure. In the case of Fabry LSD disease, the deficiency in GLA activity results in the cellular accumulation of neutral glycosphingolipids, leading to widespread blood vessel disorder with particular detriment to the kidneys, heart and nervous system.