Development of drug delivery systems based on liposomes for treatment of Fabry disease
Prof. Jan Skov Pedersen, Department of Chemistry and iNANO, is part of the new Horizon 2020 Framework Programme project “Smart-4-Fabry” (Smart multifunctional GLA-nanoformulation), funded under the action 'H2020-EU.2.1.2. - INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies – Nanotechnologies'. The project has started on January 1st, 2017, and runs for 4 year with 10 partners and a budget of 5,800,000 € of which 343,000 is allocated for Aarhus University.
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 disorders (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 nerve system.