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 |