Our RESETTM clinical trials with rese-cel (investigational CD19-CAR T cell therapy) in lupus, myositis, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris are now recruiting.

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, rese-cel, is being advanced in the RESETTM (REstoring SElf-Tolerance) Phase 1/2 clinical trials in systemic lupus erythematosus, myositis, systemic sclerosis, generalized myasthenia gravis and pemphigus vulgaris, with potential application in a broad portfolio of other autoimmune diseases in rheumatology, neurology, and other therapeutic areas.

Our Pipeline

Program T rial Preclinical Phase 1/2 Registrational (CABA-201) 4-1BB CD19-CAR T RESE T -Myositis TM RESE T -SLE TM RESE T -SSc TM RESE T -MG TM RESE T -PV TM Dermatomyositis Anti-Synthetase Syndrome Immune-mediated necrotizing myopathy Juvenile Myositis Lupus Nephritis Non-Renal SLE Skin + Organ Cohort Skin Cohort AChR-Ab pos. gMG AChR-Ab neg. gMG Mucocutaneous & mucosal pemphigus vulgaris Rese-cel 1 RESE T -MS TM Relapsing MS Progressive MS RMAT Rheumatology Neurology Dermatology Contains cohort(s) without preconditioning Pediatric indication

1Additional pipeline candidate includes MuSK-CAART for MuSK-Ab positive MG, currently being evaluated in a Phase 1 trial.
FTD – FDA Fast Track Designation received in dermatomyositis, SLE and lupus nephritis, systemic sclerosis, multiple sclerosis and generalized myasthenia gravis. RMAT – FDA Regenerative Medicine Advanced Therapy (RMAT) received in myositis and lupus.

Chimeric Antigen Receptor T cells for Autoimmunity: Rese-cel

We are developing rese-cel, 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. Rese-cel 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 rese-cel in the RESETTM Phase 1/2 clinical trials in systemic lupus erythematosus, myositis, systemic sclerosis, generalized myasthenia gravis and pemphigus vulgaris.

Learn about rese-cel