Pipeline

Clinical-stage targeted cell therapy pipeline for 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 Innovative T Cell Therapy Candidates in Autoimmune Disease

We are targeting a portfolio of autoimmune diseases with our engineered T cell therapy candidates. Our lead Chimeric Antigen Receptor T cell for Autoimmunity (CARTA) candidate, CABA-201, is being advanced in Phase 1/2 clinical trials in systemic lupus erythematosus, myositis, systemic sclerosis and generalized myasthenia gravis, with potential application in a broad portfolio of other autoimmune diseases in rheumatology, neurology, and other therapeutic areas.

Our Pipeline

CARTACAART2 CABA-201 DSG3-CAART 3 MuSK-CAART 3 Chimeric AntigenReceptor T cellsfor Autoimmunity4-1BB CD19-CAR TChimeric AutoAntibodyReceptor T cellsCABA™ PlatformProgramIndicationIND clearedIND clearedIND clearedMyositis(IIM, IdiopathicInflammatory Myopathy)1Systemic LupusErythematosus (SLE)1MuSK MyastheniaGravisMucosal PemphigusVulgarisMultiple UndisclosedIndicationsSystemic Sclerosis(SSc)1Generalized MyastheniaGravis (gMG)1DiscoveryDermatomyositisAnti-Synthetase SyndromeLupus NephritisNon-Renal SLEAChR Antibody-PositiveAChR Antibody-NegativeSevere Skin InvolvementSevere Organ InvolvementImmune-Mediated Necrotizing MyopathyPreclinicalPhase 1/2Phase 2/3

1CABA-201 is being evaluated in separate clinical trials for myositis, SLE, SSc & gMG.
2Additional CAART pipeline candidates include PLA2R-CAART in preclinical stage for PLA2R membranous nephropathy, DSG3/1-CAART in discovery stage for mucocutaneous pemphigus vulgaris & 2 undisclosed targets in discovery stage.
3Currently being evaluated in a Phase 1 trial.

Chimeric Antigen Receptor T cells for Autoimmunity: CABA-201

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 fully human CD19 binder has been clinically evaluated in a dual-CD19xCD22 CAR T in cancer through an investigator-initiated trial, where it has shown promising tolerability in ~20 patients. CABA-201 is designed to be given as a one-time infusion, with the potential to transiently, but fully, eliminate B cells, thus enabling a potential “immune system reset” and durable responses for patients with autoimmune disease. We are advancing CABA-201 in Phase 1/2 clinical trials in systemic lupus erythematosus, myositis, systemic sclerosis and generalized myasthenia gravis.

Learn about CABA-201