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0ABO2203 is an mRNA-lipid nanoparticle therapy encoding a CD19-targeting T cell engager designed to eliminate B cells with reduced cytokine release. In preclinical mouse models, this treatment successfully depleted B cells across various tissues while maintaining a favorable safety profile. A first-in-human clinical study involving three patients with treatment-refractory immune thrombocytopenia demonstrated rapid, complete peripheral and bone marrow B cell depletion. These patients achieved durable platelet recovery and reduced disease activity through six months of follow-up without experiencing severe toxicity or cytokine release syndrome. Furthermore, immunological profiling revealed a profound restructuring of the B cell and T cell repertoires, establishing this therapeutic approach as a promising strategy for resetting autoreactive immunity.
References:
Wang M, Liu T, Dai Q, et al. mRNA-encoding CD19-targeting T cell engager for refractory immune thrombocytopenia[J]. Cell, 2026.
