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BEN301 is an autologous chimeric antigen receptor regulatory T cell (CAR-Treg) therapy developed by RenJi Hospital (Shanghai Jiaotong University School of Medicine). The therapy involves engineering CD4+ T cells to express both a chimeric antigen receptor (CAR) and the transcription factor Foxp3, which is the master regulator of the regulatory T cell lineage. Unlike conventional CAR-T therapies used in oncology that are designed to eliminate target cells, BEN301 is intended to restore immune tolerance and suppress the aberrant activation of T and B cells in patients with autoimmune diseases. It is currently being evaluated in a Phase I exploratory study for the treatment of systemic sclerosis, rheumatoid arthritis, Sjogren's syndrome, and idiopathic inflammatory myopathies. Preclinical studies have also demonstrated its potential in systemic lupus erythematosus (SLE) by reducing total IgG and anti-dsDNA autoantibody production without significant treatment-related toxicities.
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