Our RESETTM clinical trials with CABA-201 (investigational CD19-CAR T cell therapy) in lupus, myositis, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris are now recruiting.
In addition to our work on autoimmune disease treatments, we are investigating the viability of CAART as an adjunct in cases where the immune system produces antibodies against current therapies (rather than against the patient’s cells). We believe our approach has the potential to destroy those antibodies by ablating B cells, similar to the method we have used to combat autoimmune disease.
FVIII-CAART is a discovery-stage candidate for Hemophilia A patients who have an anti-Factor VIII immune response. FVIII-CAART is designed to destroy the antibodies and B cells that neutralize the FVIII clotting factor – potentially restoring the effectiveness of FVIII infusions.
| CABA ™ Platform | Indication | Program | Discovery | Preclinical | Phase 1 | Phase 2/3 |
|---|---|---|---|---|---|---|
| CARTA - Chimeric Antigen Receptor T cells for Autoimmunity | Systemic Lupus Erythematous (SLE) - Lupus Nephritis¹ | CABA-201 4-1BB CD19-CAR T |
Discovery complete
|
Preclinical complete
|
Phase 1 not started
|
Phase 2/3 not started
|
| SLE without Renal Involvement¹ | CABA-201 4-1BB CD19-CAR T |
Discovery complete
|
Preclinical complete
|
Phase 1 not started
|
Phase 2/3 not started
|
|
| Multiple Undisclosed Indications | CABA-201 4-1BB CD19-CAR T |
Discovery complete
|
Preclinical in progress
|
Phase 1 not started
|
Phase 2/3 not started
|
|
| CAART2 - Chimeric AutoAntibody Receptor T cells | Mucosal Pemphigus Vulgaris | DSG3-CAART |
Discovery complete
|
Preclinical complete
|
Phase 1 in progress
|
Phase 2/3 not started
|
| MuSK Myasthenia Gravis | MuSK-CAART |
Discovery complete
|
Preclinical complete
|
Phase 1 in progress
|
Phase 2/3 not started
|
|
| PLA2R Membranous Nephropathy | PLA2R-CAART |
Discovery complete
|
Preclinical in progress
|
Phase 1 not started
|
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
|
1 In our discovery stage, we perform epitope mapping and optimize CAAR construct and design.
FVIII-CAART is designed to improve the efficacy of FVIII replacement infusions in patients with Hemophilia A.
Hemophilia A is an X-linked bleeding disorder caused by a deficiency of functional FVIII, a critical factor in blood coagulation. It affects about 1 in 5,000 male births. Severe Hemophilia A, which accounts for about 60% of all cases, is marked by FVIII levels below 1% of normal, rendering these patients vulnerable to frequent spontaneous bleeds. Currently, Hemophilia A is treated with intravenous FVIII replacement. However, nearly a third of patients who receive this therapy develop neutralizing alloantibodies against the FVIII protein. To reverse alloantibody formation, patients are given repeated, high-dose infusions of FVIII, often at a high cost and with limited clinical success. We believe FVIII-CAART could effectively eliminate FVIII-neutralizing alloantibodies and the B cells that produce them, offering a more tolerable and durable treatment option for patients with Hemophilia A and FVIII alloantibodies.
