GO-203-2C

GO‑203‑2C is a rationally designed D-amino acid cell‑penetrating peptide that targets the C-terminal subunit of mucin 1 (MUC1-C), an oncoprotein overexpressed in many human cancers including breast, prostate, lung, colon, pancreas and ovary[1][4][5]. By binding to the MUC1-C cytoplasmic domain and blocking its homodimerization required for nuclear translocation and downstream signaling[6][8], GO‑203‑2C disrupts cell-cell interactions and oncogenic signaling pathways. This leads to inhibition of tumor growth and metastasis. The drug has demonstrated antineoplastic activity in preclinical models and is under clinical investigation primarily for relapsed or refractory acute myeloid leukemia (AML)[4]. It was previously investigated for solid tumors but development was discontinued in that indication[4]. The drug was developed by Genus Oncology with Dana-Farber Cancer Institute involvement[4].

Other names

Acetyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-cysteinyl-D-glutaminyl-D-cysteinyl-D-arginyl-D-arginyl-D-lysyl-dasparaginylamide disulfide