Toleranzia’s main focus is directed towards myasthenia gravis. Myasthenia gravis is a relatively rare autoimmune nerve-muscle disease that causes severe muscle weakness and, in many cases, a difficult life situation for the affected patient.
The disease affects some 200 000 people in the EU and the US together and occurs in both sexes. It generally affects younger women (< 40 years old) and older men (> 60 years old).
Characteristic of the disease are fatigue and muscle weakness caused by an impaired transmission of nerve impulses to muscles. This is due to an immune attack directed against the acetylcholine receptor (AChR) which is the muscle relay station and the receiver of signals from the nerve. Destruction of the AChR leads to defective neuromuscular conduction of electrical impulses and a subsequent muscle weakness, which can be very serious for the patient.
Toleranzia is not aware of any current treatment that targets the root cause of the disease. Rather, today, patients with myasthenia gravis are treated with symptom-relieving therapies such as acetylcholinesterase inhibitors or immunosuppressive therapy, and, in many cases, thymectomy – a complicated surgical procedure which removes an organ, the thymus, which is part of the immune system. Common denominators of current treatments are that they have a limited effectiveness and can cause serious side effects. Therefore, there is a large unmet need for new and effective treatment options to offer patients with myasthenia gravis.
On the basis of its technology portfolio Toleranzia develops a myasthenia gravis-tolerogen. This is designed to contain protein components that are disease specific to myasthenia gravis, namely from the AChR.
With the preclinical data obtained, Toleranzia estimates that the myasthenia gravis tolerogen has the potential to become the first truly disease-modifying treatment, with curing or long-lasting therapeutic effect, to offer patients with myasthenia gravis.