WGI-0301

WGI-0301 is a lipid nanoparticle formulation of Archexin, a 20-mer phosphorothioate antisense oligonucleotide specifically designed to bind to Akt-1 mRNA. By binding to Akt-1 mRNA, it inhibits its translation and downregulates Akt-1 protein expression via RNase H-mediated degradation. This mechanism targets the PI3K/Akt pathway, which is frequently dysregulated in cancer and associated with tumor growth, survival, and angiogenesis. Preclinical studies have shown that WGI-0301 exhibits anti-tumor and antiangiogenic activity both as monotherapy and in combination with tyrosine kinase inhibitors such as sorafenib or lenvatinib. The drug is being developed primarily for advanced solid tumors including hepatocellular carcinoma (HCC), where it has received orphan drug designation in the US[3][5][6][7].

Brand names

Mychexin

Other names

AKT-1 antisense oligonucleotide WGI-0301AKT1 antisense oligonucleotide WGI-0301AKT 1 antisense oligonucleotide WGI-0301