V001-BCMA

V001-BCMA is an experimental in vivo CAR-T cell therapy designed to treat refractory autoimmune diseases. Developed by Tongji Hospital, it employs a third-generation, non-replicating, self-inactivating lentiviral vector. The vector's envelope is engineered to specifically recognize and bind to T cells in the bloodstream. Upon binding, it introduces a chimeric antigen receptor (CAR) gene under the control of a T cell-specific promoter. This allows the patient's own T cells to express a CAR targeting B-cell maturation antigen (BCMA), effectively creating CAR-T cells in vivo. These cells then target and eliminate BCMA-expressing plasma cells, which are responsible for producing pathogenic autoantibodies in diseases like systemic lupus erythematosus and systemic sclerosis.

Other names

Targeted BCMA In-vivo LV Injection