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USP6 mRNA LNP is an experimental immunotherapy consisting of messenger RNA (mRNA) encoding the Ubiquitin-specific protease 6 (USP6) protein, encapsulated in lipid nanoparticles (LNPs) for delivery. Developed by Merlin Biotech in collaboration with the Blumberg Institute and academic institutions, the drug is designed for intratumoral administration. USP6 is a hominid-specific protein that, when expressed in tumor cells, upregulates several immunostimulatory chemokines (such as CXCL9, CXCL10, and CCL5) and surface ligands (including CD112, MICA/B, ICAM1, and MHC Class I) that promote the recruitment and activation of cytotoxic immune cells. It also increases tumor cell sensitivity to immune-mediated apoptosis via Type I/II interferons and TRAIL. Preclinical data presented at AACR 2023 demonstrated that intratumoral injection of USP6 mRNA LNPs inhibits the growth of both local and distal tumors in murine models of Ewing sarcoma and acute myeloid leukemia.
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