Our RESETTM clinical trials with CABA-201 (investigational CD19-CAR T cell therapy) in lupus, myositis, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris are now recruiting.

Patients

Autoimmune Diseases

Autoimmune diseases occur when the immune system becomes misdirected to the healthy cells and tissues of the body, causing inflammation and damage. Normally, the immune system is responsible for protecting the body against “foreign invaders,” such as bacteria and viruses. However, in autoimmune disease, the immune system incorrectly views healthy tissues as foreign, leading to damage and dysfunction of the affected organs or tissues.

There are dozens of autoimmune diseases that can affect many parts of the body, including the joints, skin, internal organs, and nervous system. Some examples include systemic lupus erythematosus, rheumatoid arthritis, and multiple sclerosis. Treatment typically involves managing the symptoms and suppressing the immune system to prevent further damage.

How are B cells involved in autoimmune diseases?

B cells are essential components of the body’s immune system. They manufacture specialized proteins called antibodies that circulate in the blood and bind like a lock-and-key to the surfaces of bacteria and viruses, the foreign invaders responsible for causing disease. They also recruit and encourage other immune cells to respond to the foreign invaders and to secrete factors that increase inflammation.

In autoimmune diseases, B cells contribute to causing disease by initiating or continuing the self-directed immune response. In some cases, B cells develop receptors that mistakenly target healthy tissues and cells. Once activated, these B cells multiply and produce autoantibodies, or antibodies against self-proteins, that attack healthy cells. The disease-causing B cells may also engage other immune cell types in attacking healthy tissue. These B cells cause certain types of autoimmune diseases by perceiving healthy tissues as foreign and mounting an immune response.

Is there a cure for autoimmune diseases?

There is currently no cure for autoimmune diseases. Current treatment options for autoimmune diseases typically involve generalized immune suppression through corticosteroids as well as immunosuppressant medications and biologics. All of these current methods impair or destroy healthy B cells and/or other immune cells as well as pathogenic ones, weakening the patient’s overall immune function, potentially putting them at risk for infection and impairing their response to vaccines. In general, these drugs require long-term administration and may have life-threatening side effects. The ideal therapy in autoimmune diseases with B cell involvement would cause elimination of disease-causing B cells with restoration of the normal immune system, enabling an “immune reset,” restoring the body’s immune system to its normal function of fighting foreign invaders, not healthy tissues.

Autoimmune Diseases We Target

We are developing T cell therapy candidates in the following disease areas:

Systemic Lupus Erythematosus (SLE)

SLE is a chronic autoimmune disease that occurs primarily in young women and causes a wide range of clinical manifestations that may become life-threatening. Lupus nephritis is a serious complication of SLE that carries risk of renal failure and mortality. Our RESET-SLETM Phase 1/2 trial of CABA-201 is currently ongoing.

Myositis

Myositis is an autoimmune disease that typically occurs in middle age and is more common in women. Myositis causes inflammation and severe muscle weakness, in addition to characteristic skin manifestations and internal organ involvement that may become severe. Our RESET-MyositisTM Phase 1/2 trial of CABA-201is currently ongoing.

Systemic Sclerosis (SSc)

SSc is a chronic autoimmune disease that typically occurs in middle age and is more common in women. SSc causes progressive fibrosis and scarring of the skin and internal organs that can be potentially fatal. Our RESET-SScTM Phase 1/2 trial of CABA-201 is under initiation.

Generalized Myasthenia Gravis (gMG)

gMG is an autoimmune disease with potential for profound impact on quality of life that occurs in both younger and older individuals. gMG is caused by autoantibodies that lead to potentially severe muscle weakness and risk for episodes of respiratory failure. Our RESET-MGTM Phase 1/2 trial of CABA-201 in gMG is under initiation.

Pemphigus Vulgaris (PV)

PV is an autoimmune disease that causes blisters on the skin and mucous membranes. The DesCAARTes Phase 1 trial in mucosal PV is ongoing.

Muscle-Specific Kinase Myasthenia Gravis (MuSK MG)

MuSK MG is an autoimmune disease that causes risk of debilitating and life-threatening muscle weakness. The MusCAARTes Phase I trial in MuSK MG is ongoing.

Phase 1/2 Trial of CABA-201 in SLE

We are currently recruiting patients with SLE & LN for our RESET-SLETM Phase 1/2 trial.

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