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48 Monate

Smart functional GLA nanoformulation for Fabry disease

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.

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European Commission

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


Project team

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