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.

Is there a cure for B cell-mediated autoimmune diseases?

There is currently no cure for B cell-mediated autoimmune diseases. 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.

Autoimmune 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 Cell Therapy Pipeline

DesCAARTes™ Phase 1 Trial

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

Tell Me More About the Clinical Trial

Our Technology

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

Learn More About Our Cell Therapy Technology