TOL3: A Novel Drug for the Autoimmune Disease ANCA Vasculitis
| Reference number | |
| Coordinator | Toleranzia AB |
| Funding from Vinnova | SEK 1 992 648 |
| Project duration | April 2020 - December 2021 |
| Status | Completed |
| Venture | Innovation projects in enterprises |
| Call | Innovation projects in small and medium-sized companies - autumn 2019 |
Important results from the project
The purpose and goal to develop a disease modifying therapy (TOL3) for treatment of ANCA vascu-litis has come a long way. The results generated in this project provide a foundation for moving forward with TOL3 development and validate Toleranzia’s tolerogen technology for treatment of autoimmune diseases.
Expected long term effects
A rat model of MPO-ANCA vasculitis was successfully established. Several variants of TOL3 were designed and the one that gave highest expression in E. coli was manufactured at small scale and tested in the animal model showing a positive trend towards amelioration of disease. An immunogenicity analysis of TOL3 in silico showed no increased risk in humans. Regarding IP, the FTO analysis did not identify anything that could limit TOL3’s development and a patent application for TOL3 was filed. The results generated in this project are very encouraging and support further TOL3 development.
Approach and implementation
Toleranzia will continue to work towards achieving proof of concept with TOL3. The design of the TOL3 generated in this project was based on the very scarce available knowledge of patients´ reac-tivity to MPO, the autoantigen in ANCA. The next step is to investigate all MPO epitopes that ANCA patients present and react to and redesign TOL3 to contain a more complete set of disease-specific epitopes, which will potentiate its tolerogenic capacity.