Product name
GBA2 - Therapy of glycolipid storage diseases from Universities Aachen and TexasSummary
enzyme replacement therapy especially for the severe forms of Gaucher`s diseaseOrganization name
ProvendisProfile
Glycosphingolipids represent a particularly complex class of glycolipids. The high structural diversity is generated by an equally elaborate network of metabolic enzymes. Loss of degradative enzymes causes glycolipid storage diseases, such as Gaucher`s disease, which results from a functional impairment or loss of the glycosylceramidase GBA1. The second glycolipid hydrolase, GBA2, is also involved in this storage disease. GBA2 does not show sequence identity to GBA1 and is expressed in different tissues and subcellular compartments.
Substitution of GBA2 can be used to treat glycolipid storage diseases and to support or replace the function of GBA1 in such diseases especially in the severe forms of Gaucher`s disease where GBA1 is not utilized.
Further, the invention allows drug screening based on GBA2 knock-out mouse models and genetic expression systems. The knock-out mice are currently characterized concerning physiology as well as behaviour.
Commercial opportunities
The incidence of Gaucher`s disease is around 1:40000 to 1:60000 worldwide, among turkish and jewish people it is even around 1:1000. The treatment expenses in Germany run into hundreds of thousands of EURO per year per patient. The GBA2 enzyme can be utilized as an alternative or additional enzyme replacement therapy especially for the severe forms of Gaucher`s disease. Side effects of the sole GBA1 enzyme therapy may thus be reduced. The GBA2 enzyme can be produced easily in its active form. Substitution of GBA2 may also be an alternative for patients who do not benefit from the use of inhibitors of the synthesis of glycosylceramides.
The invention provides the use of GBA2 as therapeutic for storage diseases as well as the use of screening models with GBA2 knock-out mice for the development of new therapeutics.
Current status
A German and a PCT application are pending. We offer a patent license, a research collaboration with licensing option as well as knock-out mice and transgenic cells. Convincing in vitro and in vivo data for expression of GBA2 are available.
Benefits
- Enzyme replacement in glycolipid strage diseases with potential orphan drug status
- New therapeutic strategies for Gaucher's Disease
- Validated GBA2 mouse models
- Easy to express enzyme for cheap production
Contact
Dipl.-Biol. Kordula Kruber
Tel: +49 208 94 105 30
kk(at)provendis.info
About PROvendis GmbH
PROvendis offers companies certified and patented innovations from 24 North Rhine-Westphalian universities. The core function of the enterprise is to market patentable and commercially exploitable inventions. PROvendis GmbH was founded in 2001 as a wholly owned subsidiary of Zenit GmbH, a management consultancy firm in cooperation with the state of North Rhine-Westphalia. Since January of 2002, PROvendis has been the service provider for the universities of the state of North Rhine-Westphalia. Within the scope of the BMBF commercialization campaign, the Federal Ministry for Education and Research (BMBF) supports the activities of PROvendis.
Description File
TO_GBA2_1188.pdf
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