Autoimmune Disease is caused when the normal control mechanisms of the human immune system break down, resulting in immune attack on the body’s cells and tissues leading to a wide range of disease pathologies.

Autoimmune Diseases include Autoimmune Myopathies, Crohn’s Disease, Graft-Versus Host Disease, Multiple Sclerosis, Myasthenia Gravis, Pemphigus Vulgaris, Psoriasis, Rheumatoid Arthritis, Systemic Lupus Erythematosus, Systemic Sclerosis, Sjorgren’s Syndrome, Type 1 Diabetes, among others. These diseases can start early and last over a lifetime, resulting in decades of suffering and disability.

Proliferation of activated lymphocytes is a normal component of the immune reaction towards pathogens, but in autoimmune and inflammatory diseases the proliferation of activated lymphocytes is deleterious. Accordingly, several classes of immunosuppressive agents have been developed to inhibit and control these excessive and pathological responses. However, most of these known inhibitors are associated with toxicity due to their non-specific broad effects that limit their efficacy and use. More recent treatments acting specifically on a particular mediator of the disease (e.g. cytokines) have shown more limited side effects but leave a high proportion of non-responding patients without therapeutic solution. There is therefore a great need for novel safe and effective treatments for Autoimmune Diseases.

A goal in the development of new drugs for auto-immune diseases is to look for agents with novel mechanisms of action that provide specific modulation of the immune system rather than global immunosuppression. Step Pharma’s CTPS1 inhibitors have the potential to achieve selective modulation of activated lymphocytes, translating to better safety and efficacy. These inhibitors may also bring benefit to patients suffering from selected cancers.