A unique tolerance inducing therapy for the treatment of the Orphan disease Myasthenia Gravis (MG).

Purpose and goal

MG is an autoimmun disease with a large unmet medical need for a better therapy. Through the Eurostars project Toleranzia has been able to evaluate the effect on human patient cells in vitro, develop a preclinical program as well as a synopsis for a clinical trial. The first large scale production has been performed which places us a long way down the road towards our goal to show the safety and efficacy of the therapy in humans. The project has given us increased knowledge of the tolerogen and has lead to further development of the company as well as the employment of two more persons.

Expected results and effects

The project meant that we now have a detailed preclinical program, an extended network and increased knowledge in preparation for the upcoming development steps. The staff has increased from 2 to 4 FTE. The in vitro studies on human cells were unfortunately not as evident as we hoped for, which might be explained by the complexity of the immune system and the difficulties of recreating full functionality in vitro. In the analysis on cells from healthy volunteers, lacking the autoreactive cells on which our tolerogen is supposed to act, no general biomarker for tolerance could be identified

Planned approach and implementation

The planned activities have been carried out with satisfactory results. Some delays have occurred, depending on new devlopement and studies in biological systems where the outcome is not always as expected. When problems occured, Toleranzia and its partners, worked together to solve the problems and the project was finished in the best way possible. This project have taken Toleranzia to the point where we stand today with a strong team with the competence and resources to take the us further and successfully develop a tolerance inducing therapy for the treatment of autoimmune diseases.