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LLE-aCD123 is an investigational mRNA-based therapeutic developed by InnoRNA Biotech for the treatment of acute myeloid leukemia (AML). The drug utilizes InnoRNA's proprietary Lipid-Like Envelope (LLE) delivery system to encapsulate mRNA encoding a bispecific T-cell engager (BiTE). This BiTE is designed to simultaneously target CD123 (Interleukin-3 receptor alpha), which is highly expressed on AML blasts and leukemic stem cells, and CD3 on the surface of T-cells. Upon administration, the mRNA is translated in vivo, leading to the production of the bispecific antibody which facilitates the formation of an immunological synapse between cytotoxic T-cells and malignant cells, resulting in targeted cell lysis. This approach aims to combine the potent efficacy of bispecific antibodies with the transient expression and potentially improved safety profile afforded by mRNA-based delivery.
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