Project: Allosteric chaperones to treat rare lysosomal storage disorders

Acronym CHAPERONE (Reference Number: 113321)
Duration 01/10/2019 - 03/10/2022
Project Topic 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.
Network Eurostars 2
Call Eurostars Cut–off 11

Project partner

Number Name Role Country
1 GT Gain Therapeutics SA Coordinator Switzerland
2 NeuroSys Partner France
3 Fondazione per l’Istituto di Ricerca in Biomedicina (IRB) Partner Switzerland