Patients

B Cell-Mediated 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 “foreign invaders,” or pathogens, such as bacteria and viruses. The way B cells detect and respond to these foreign invaders is quite elegant: each B cell has one type of millions of potential types of receptor proteins on its surface. As a B cell floats around the body, it eventually collides with a protein on the surface of a pathogen that connects to its receptor. The B cell binds with this perceived pathogen and gets activated, causing the B cell to divide. The dividing B cells continue to produce the same receptors that bound to the pathogen, some of which remain on the surface of the B cells, while others are released into the blood as antibodies.

In rare 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. These autoantibodies can cause certain types of autoimmune diseases, called B cell-mediated autoimmune diseases, because they are caused by the B cells mistakenly perceiving healthy tissues as foreign and mounting an immune response.

Current treatment options for B cell-mediated autoimmune diseases involve generalized immune suppression through corticosteroids as well as immunosuppressant medications and biologics. All of these current methods impair or destroy healthy B cells as well as pathogenic ones, weakening the patient’s overall immune function. In general, these drugs require long-term administration and may have life-threatening side effects. The ideal therapy in autoimmune diseases would completely and specifically eliminate only the disease-causing B cells while sparing the healthy immune cells that protect against infection by foreign invaders, without requiring long-term treatment.

Diseases We Target

We have announced that we are developing CAART candidates in the following disease areas:

Pemphigus Vulgaris (PV)

PV is an autoimmune disease that causes blisters on the skin and mucous membranes.

Muscle-Specific Kinase Myasthenia Gravis (MuSK MG)

MuSK MG is an autoimmune disease that causes debilitating and life-threatening muscle weakness.

Phospholipase A2 Receptor-Associated Membranous Nephropathy (PLA2R MN)

PLA2R-associated MN is an autoimmune disease that causes nephrotic syndrome and may lead to kidney failure.

Hemophilia A with Factor VIII Alloantibodies

Hemophilia A with Factor VIII alloantibodies is a bleeding disorder in which an immune response is provoked to the essential clotting factor used to treat the disease.

Our Pipeline

DesCAARTes™ Phase 1 Trial

We are currently recruiting patients with mPV for our Phase 1 trial.

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Our Technology

Interested to see behind the curtain? Find out more about our CAART cell therapy platform.

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