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TOA02 is a genetically modified, replication-competent oncolytic adenovirus engineered for tumor specificity through regulation by a modified human telomerase reverse transcriptase (hTERT) promoter, which includes inserted E2F-binding sites. It is designed to selectively replicate in tumor cells with high telomerase activity and retinoblastoma (Rb) pathway deficiency. TOA02 additionally expresses human granulocyte macrophage colony-stimulating factor (GM-CSF), enhancing antitumor immune responses by promoting dendritic cell and macrophage maturation. Strong antitumor efficacy has been demonstrated in vivo in murine xenograft models of human cancers, with favorable safety and low toxicity observed in non-human primates.
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