Immunomodulating therapy for the treatment of the rare disease Myasthenia Gravis.

Purpose and goal

Toleranzia want to develop a specific immunomodulating therapy for treatment of the rare, autoimmune disease MG, that lacks specific treatment today. The aim of this project was to obtain new knowlegde that would support an increased interest in Toleranzia. With the financial support from VINNOVA therapeutic effect in an animal model of MG has been obtained, as well as preliminary data on tolerance induction in human cells ex vivo. Orphan Drug Designation applications have been sent to the EMA and FDA. The approved Eurostars application will assure the continuation of the project.

Results and expected effects

The project has added a lot of new knowledge to Toleranzia which has stregthened the position of the company. New collaborators have been identified and contacts with new investors are advanced. An emission of 1,5 MSEK was fully signed and closed in October and a new emission has been opened that will close in January. With the financial support from VINNOVA Toleranzia have taken the next step towrds developing a new and unique therapy for the treatment of autoimmun diseases.

Approach and implementation

The following activities have been performed: 1. EAMG animal studies with proven effect of therapeutic treatment. 2. In vitro studies on human cells indicating tolerance induction. 3. Production of fusion proteins. 4. Process development. 5. Business development & commersialisation have identified several new investors. 6. Applications for Orphan Drug Designations have been sent to FDA and EMA. 7. Project management 8. The IP situation has been analysed and IP strategy has been developed.