OTX-2002

OTX-2002 is a first-in-class, programmable mRNA therapeutic known as an Omega Epigenomic Controller (OEC). It is designed to downregulate MYC expression pre-transcriptionally through epigenetic modulation. The drug consists of a bicistronic mRNA encoding for ZF-DNMT and ZF-KRAB proteins, encapsulated in a clinical lipid nanoparticle (LNP) formulation. By creating epigenetic marks at structural and regulatory elements within the insulated genomic domain of the MYC gene, OTX-2002 achieves highly specific and durable suppression of MYC expression. This mechanism inhibits tumor cell viability while sparing normal cells. OTX-2002 is being developed primarily for hepatocellular carcinoma (HCC), but it is also under investigation for other solid tumors associated with dysregulated MYC oncogene activity[1][3][5][6].

Other names

Omega Epigenomic Controller