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CHAPERONE - Allosteric chaperones to treat rare lysosomal storage disorders

People

 

Molinari M.

(Responsible)

Abstract

The consortium will deliver proof-of-concept (PoC) data for a novel class of therapeutic molecules to treat rare lysosomal storage disorders (LSD), for which no cure exists. A computational platform that identifies unique allosteric enzyme binding sites has generated novel compounds to restore the function of LSD-causing defects in enzymes. During the project the efficacy to restore enzyme function will be validated resulting in a data package including efficacy and mode-of-action.

Additional information

Acronym
CHAPERONE
Start date
01.10.2019
End date
31.10.2022
Duration
37 Months
Funding sources
Confederazione svizzera
Status
Active
Category
European and International Programmes / Horizon 2020 / Collaborative projects - ICT