Scientists at the Sirius University of Science and Technology will develop innovative mRNA drugs for targeted therapy of severe autoimmune diseases that currently have no specific treatment. The team will focus on creating mRNA drugs that selectively target pathological immune cells. The initial priority is rare but severe conditions such as myasthenia gravis – an autoimmune disorder affecting neuromuscular junctions – and pemphigus vulgaris, which causes blistering of the skin and mucous membranes.
To treat these diseases, the researchers will use a new technology based on CAAR-T cells. The approach resembles established CAR-T therapy, but instead of targeting tumours, it targets “harmful” B-cells that attack the body’s own tissues. CAAR-T cells can find and destroy only pathological B-cells while leaving healthy ones untouched, the scientists said. This means the patient’s immune system is not completely suppressed and retains its ability to fight infections.
The second part of the project focuses on developing mRNA cocktails for inflammatory bowel diseases such as Crohn’s disease. These drugs will be delivered directly to the gut using tiny lipid nanoparticles, the researchers said. Once in place, they will produce antibodies or antibody fragments that block inflammation-causing substances. The therapy may also activate special regulatory T-cells that restore immune balance in the patient’s body.
The third area of work is tackling autoimmune diabetes. The research team will develop specialised lipid nanoparticles capable of delivering mRNA‑based drugs directly to the pancreas. In the future, they plan to create tolerogenic vaccines that “train” the immune system not to attack the body’s own pancreatic cells, thereby helping to protect the organ from destruction.
The development of such drugs will open new possibilities for treating severe autoimmune diseases and significantly improve the quality of life for thousands of patients across the country, the scientists stressed.