The immune system is programmed to avoid attacking the body’s own cells and tissues in a mechanism known as self-tolerance. The negative regulators of the immune system, known as immune checkpoints, play a critical role in the maintenance of self-tolerance. In autoimmune diseases self-tolerance is compromised and the immune system mistakenly attacks the body’s own tissues. Autoimmune diseases are characterized by hyperactive immune responses that target self-antigens, leading to chronic inflammation and targeted destruction of tissues and organs. An autoimmune disease can be limited to a single organ or may affect multiple organs and tissues. Autoimmune diseases affect millions worldwide, who may suffer from symptoms ranging from mild rashes to life-threatening conditions when major body organs are affected. Autoimmune diseases are a leading cause of disability and death.
There is currently no cure for autoimmune diseases, and existing therapeutic approaches rely mostly on general suppression of the immune system. These treatments are symptomatic and can compromise the capacity of the immune system to fight infectious diseases and malignancies. Advances in the treatment of autoimmune diseases include biologic drugs that specifically target inflammation mediators, known as cytokines that cause chronic inflammation. Significant unmet need remains as many autoimmune diseases have no or few treatment options, and in others, patients have limited benefit from existing therapies.
Activation of immune checkpoints to control the immune system and re-establish immune tolerance is a new paradigm in the development of therapies for autoimmune diseases. Compugen’s novel immune checkpoints have shown therapeutic efficacy in models of autoimmune diseases, and CGEN-15001 is our lead product candidate in this field.