Pipeline

Clinical-stage cell therapy pipeline for B cell-mediated autoimmune diseases

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 CAAR T cell therapy candidates to treat B cell-mediated autoimmune diseases. Our lead product candidate, DSG3-CAART, which is being evaluated in the DesCAARTes™ trial, targets B cells that cause mucosal pemphigus vulgaris (mPV), a life-threatening autoimmune blistering disease affecting the mucous membranes.

Our Pipeline

Other

Therapeutic Area Indication Program Discovery Preclinical Phase 1 Phase 2/3
Dermatology Mucosal Pemphigus Vulgaris DSG3-CAART
Discovery complete
Preclinical complete
Phase 1 in progress
Phase 2/3 not started
Mucocutaneous Pemphigus Vulgaris DSG3/1-CAART
Discovery in progress
Preclinical not started
Phase 1 not started
Phase 2/3 not started
Neurology MuSK Myasthenia Gravis MuSK-CAART
Discovery complete
Preclinical in progress
Phase 1 not started
Phase 2/3 not started
Nephrology PLA2R Membranous Nephropathy PLA2R-CAART
Discovery complete
Preclinical in progress
Phase 1 not started
Phase 2/3 not started
Hematology Hemophilia A with FVIII Alloantibodies FVIII-CAART
Discovery in progress
Preclinical not started
Phase 1 not started
Phase 2/3 not started

Two additional undisclosed discovery programs added to our pipeline through our Sponsored Research Agreement expansion with the University of Pennsylvania are not shown.

1 In our discovery stage, we perform epitope mapping and optimize CAAR construct and design.

DSG3-CAART for Mucosal Pemphigus Vulgaris

Our lead product candidate, DSG3-CAART, targets 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.

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.

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 blistering skin disease.

Learn about DSG3/1-CAART for mcPV

FVIII-CAART for Hemophilia A with Factor VIII Alloantibodies

Sometimes B cells produce antibodies that attack essential drug therapies, leaving patients who have these alloantibodies with few treatment options. We are engineering CAARs to selectively destroy these alloantibody-producing B cells, enabling life-saving treatment to potentially continue.

Learn about FVIII-CAART for Hemophilia A with Inhibitors