First small molecule drug inhibitor of microRNAs: Treatment for systemic lupus erythematosus
| Reference number | |
| Coordinator | Redoxis AB |
| Funding from Vinnova | SEK 2 089 053 |
| Project duration | April 2019 - September 2022 |
| Status | Completed |
| Venture | Eurostars |
Important results from the project
MicroRNA miR-155 regulates several inflammatory genes and is overexpressed in SLE patients. Studies have shown that inhibition of miR-155 can be an efficient and safe treatment for SLE. The aim of the study was to develop a "first-in-class" drug for the treatment of SLE by inhibiting miR-155 and TNFa. The project has largely reached its objective and a small number of "lead compounds" have been selected through various in vitro and in vivo models, and can be used for continued drug development
Expected long term effects
During the project, we have developed an inflammatory in vitro model to be able to screen and evaluate substances based on inhibition of miR-155, TNFa and toxicity. About 150 substances have been evaluated in this model and 3 lead compounds have been selected. PK and efficacy studies in inflammatory models have been performed. Finally, these "lead compounds" were tested in an SLE mice model with promising initial results.
Approach and implementation
Within the project, we have developed an inflammatory in vitro model to be able to screen and evaluate substances based on the inhibition of miR-155, TNFa and toxicity. The substances have been designed and synthesized by our project partner and have then been evaluated for effect by us. Together, we have analyzed the results and selected a smaller number of "lead compounds" on which we have then run PK, safety and efficacy studies in mice.