Innovating Works

Sphingolead

Financiado
Development of a potent dual GCS GBA2 inhibitor as a best in class Gaucher thera...
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 replacem... 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. ver más
28/02/2018
ULEI
150K€
Duración del proyecto: 18 meses Fecha Inicio: 2016-08-11
Fecha Fin: 2018-02-28

Línea de financiación: concedida

El organismo H2020 notifico la concesión del proyecto el día 2018-02-28
Línea de financiación objetivo El proyecto se financió a través de la siguiente ayuda:
ERC-PoC-2015: ERC Proof of Concept Grant
Cerrada hace 9 años
Presupuesto El presupuesto total del proyecto asciende a 150K€
Líder del proyecto
UNIVERSITEIT LEIDEN No se ha especificado una descripción o un objeto social para esta compañía.
Perfil tecnológico TRL 4-5