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 ® RESE T -SLE TM RMAT RESE T -SSc TM RMAT RESE T -MG TM RESE T -PV TM Dermatomyositis Antisynthetase 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 Pemphigus Vulgaris Rese-cel 1 RMAT Rheumatology Neurology Dermatology Contains cohort(s) without preconditioning Pediatric indication 1

RESET™ – REstoring SElf-Tolerance; Ab – Antibody; AChR – Acetylcholine receptor; gMG – Generalized myasthenia gravis; PV – Pemphigus vulgaris; SLE – Systemic lupus erythematosus; SSc – Systemic sclerosis

1. Myositis patients can also be treated by neurologists or dermatologists; lupus nephritis patients can also be treated by nephrologists.

FDA Fast Track Designation received in dermatomyositis, SLE and lupus nephritis, systemic sclerosis, generalized myasthenia gravis and multiple sclerosis.

FDA Regenerative Medicine Advanced Therapy (RMAT) received in myositis, SLE, LN and systemic sclerosis.

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