RAC2 G12R mRNA-LNP

RAC2 G12R mRNA-LNP is an experimental mRNA-based therapeutic consisting of mRNA encoding the constitutively active G12R variant of the Ras-related C3 botulinum toxin substrate 2 (RAC2) protein, encapsulated in lipid nanoparticles (LNPs). This strategy repurposes a deleterious mutation associated with severe combined immunodeficiency (SCID) as a therapeutic agent for hematological malignancies. The RAC2 G12R variant disrupts nucleotide metabolism and induces rapid cell death via a necroptosis-like signaling pathway. Developed by researchers at the Imagine Institute and INSERM, the therapy is being investigated for the treatment of acute myeloid leukemia (AML) and other hematologic disorders by selectively inducing apoptosis in malignant cells.