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Development of a potent dual GCS/GBA2 inhibitor as a best in class Gaucher therapeutic

Total Cost €


EC-Contrib. €






Project "Sphingolead" data sheet

The following table provides information about the project.


Organization address
address: RAPENBURG 70
city: LEIDEN
postcode: 2311 EZ

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Netherlands [NL]
 Total cost 150˙000 €
 EC max contribution 150˙000 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2015-PoC
 Funding Scheme ERC-POC
 Starting year 2016
 Duration (year-month-day) from 2016-09-01   to  2018-02-28


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITEIT LEIDEN NL (LEIDEN) coordinator 150˙000.00


 Project objective

Gaucher disease (GD) is an autosomal recessive disorder caused by deficient activity of acid glucosylceramidase (GBA1). Most patients feature a non-neuronopathic (type 1) course of disease and are treated by either enzyme replacement therapy (ERT – administration of recombinant GBA1) or substrate reduction therapy (SRT – treatment with the iminosugar, Zavesca or the ceramide mimic, Eliglustat – inhibitors of glucosylceramide synthase (GCS)). These therapeutic agents do not prevent neurological symptoms in patients suffering from neuronopathic Gaucher forms (types 2/3) and there is thus an unmet medical need for these patients. Potent GCS inhibitors reduce glucosylceramide (GlcCer), the substrate of GBA and the storage material in Gaucher, and also higher glycosphingolipids including gangliosides and globosides, the causative storage materials in Tay-Sachs disease, GM1 gangliosidosis and Fabry disease. We have found a highly attractive lead, BiPheDNM 7 (Figure C below), which we plan to further develop towards a phase I/II clinical candidate. This proof of concept project will result in a packet comprising this lead molecule, accompanied by all the relevant data and tools to start phase I/II clinical development together with a pharmaceutical partner.

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The information about "SPHINGOLEAD" are provided by the European Opendata Portal: CORDIS opendata.

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