Cell Therapy Clinical Trial Pipeline

In certain autoimmune diseases, pathogenic B cells secrete autoantibodies, or antibodies that mistakenly target healthy host proteins, which are responsible for causing disease.

Advancing an Innovative Collection of Autoimmune Therapy Candidates

We are developing a portfolio of engineered T cell therapy candidates to treat autoimmune diseases. Our lead Chimeric Antigen Receptor T cell for Autoimmunity (CARTA) candidate, CABA-201, is being developed for systemic lupus erythematosus as well as other severe autoimmune diseases.

Our lead Chimeric AutoAntibody Receptor T (CAART) cell therapy product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ trial, targeting B cells that cause mucosal pemphigus vulgaris (mPV), a life-threatening autoimmune blistering disease affecting the mucous membranes. The MusCAARTes™ trial of MuSK-CAART in MuSK-associated myasthenia gravis is ongoing, with the development strategy accelerated through learnings informed by the DSG3-CAART experience. We also have several preclinical and discovery-stage targeted cell therapy candidates under development, all created with the vision of improving the lives of patients with autoimmune diseases.

Our Pipeline

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¹Being evaluated in a single clinical trial for CABA-201.
²Additional disease targets in discovery stage in our CAART pipeline portfolio include two undisclosed indications.

CABA-201 for B Cell-Mediated Autoimmune Diseases

We are developing CABA-201, a fully human CD19 chimeric antigen receptor (CAR) T cell therapy containing a 4-1BB co-stimulatory domain, for autoimmune diseases with high unmet need. The initial indication for development is systemic lupus erythematosus (SLE), a chronic autoimmune disease that occurs primarily in young women and causes a wide range of clinical manifestations that may become life-threatening. CABA-201 is also in preclinical development for other autoimmune diseases.

Learn about CABA-201

DSG3-CAART for Mucosal Pemphigus Vulgaris

Our lead CAART product candidate, DSG3-CAART, is an engineered T cell therapy targeted to the B cells that cause mucosal pemphigus vulgaris (mPV), a life-threatening autoimmune blistering disease that leads to severe erosion and compromise of mucous membranes. It is currently being evaluated in patients with mucosal pemphigus vulgaris in the DesCAARTes™ Phase 1 clinical trial.

Learn about DSG3-CAART for mPV

MuSK-CAART for MuSK Myasthenia Gravis

Muscle-specific Kinase Myasthenia Gravis (MuSK MG) is an autoimmune disease that leads to disabling muscle fatigue, motor impairment, and episodes of respiratory failure. We are developing MuSK-CAART to target the B cells that cause this disease, and the candidate is currently being evaluated in patients with MuSK MG in the MusCAARTes™ Phase 1 clinical trial.

Learn about MuSK-CAART for MuSK MG

PLA2R-CAART for PLA2R-Associated Membranous Nephropathy

Phospholipase A2 Receptor-Associated Membranous Nephropathy (PLA2R MN) is an autoantibody-mediated glomerular disease that causes nephrotic syndrome and may lead to kidney failure. We are engineering PLA2R-CAART to selectively deplete the B cells that produce these disease-causing autoantibodies.

Learn about PLA2R-CAART for PLA2R MN

DSG3/1-CAART for Mucocutaneous Pemphigus Vulgaris

We are developing DSG3/1-CAART to target mucocutaneous pemphigus vulgaris (mcPV), a painful blistering disease that affects both the skin and mucous membranes and predisposes patients to potentially life-threatening infections.

Learn about DSG3/1-CAART for mcPV