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LNP-CTNNB1 is a lipid nanoparticle (LNP) formulation encapsulating siRNA that specifically targets the CTNNB1 gene, which encodes β-catenin[1][2][3][4]. This agent is designed for RNAi-mediated gene knockdown in hepatocellular carcinoma (HCC) models bearing CTNNB1 mutations. Mechanistically, it suppresses β-catenin signaling, resulting in reduced expression of key Wnt/β-catenin target genes (such as Glul and Ccnd1), decreased tumor cell proliferation, and increased tumor cell death, reprogramming both tumor cells and the immune microenvironment to enhance antitumor response[1][2][3][4]. Preclinical data demonstrates efficacy as a monotherapy and in combination with immune checkpoint inhibitors (ICI), extending survival and restoring antitumor immune function with durable tumor suppression in models of CTNNB1-mutated HCC[1][2][3][4]. The molecule is designed for systemic administration (intravenous) in preclinical models[1][4]. Developer details are not explicitly stated in the sources.
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