A new medical therapy for children with severe muscular disease
| Reference number | |
| Coordinator | MEROZYNE THERAPEUTICS AB |
| Funding from Vinnova | SEK 500 000 |
| Project duration | May 2016 - December 2016 |
| Status | Completed |
Purpose and goal
Merozyne Therapeutics is working to develop a pharmacological treatment of muscle disease MDC1A. In this process, work has focused on establishing various so-called screening methods to quickly evaluate new chemical substances. These methods are based on relevant enzymatic and cellular processes. With the support of Vinnova these screening methods are now established in the project.
Expected results and effects
The project now has acquired and synthesized a group of reference kinase inhibitors. These inhibitors have been used for establishing, testing and validating a enzymatic and a cellular assay. Furthermore, the substances are used to obtain important and vital chemical information that will be used in the further project development, with a focus on developing new chemical substances that have the potential to be developed into pharmaceutical drugs.
Planned approach and implementation
In the future development of a pharmaceutical drug against muscle disease MDC1A it is important that processes are in place that can be used methodically to develop new chemical drug candidates. Such processes include both enzymatic and cellular assays. Work with chemical structures, both in the laboratory and theoretically, have also been carried out and important information has been obtained. Taken together, the necessary drug development processes have now been established in the project.