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Vocimagene amiretrorepvec is an investigational gene therapy agent delivered as a retroviral replicating vector (RRV) encoding the yeast cytosine deaminase (yCD) gene. Upon injection into tumor tissue, the vector selectively infects dividing cells—primarily cancer cells—and causes them to express cytosine deaminase. When the prodrug flucytosine (5-fluorocytosine, 5-FC) is administered systemically, it is converted by cytosine deaminase within infected tumor cells into the chemotherapeutic agent 5-fluorouracil (5-FU), resulting in local cytotoxicity and immune activation. This approach aims to achieve targeted chemotherapy within tumors while minimizing systemic toxicity. The therapy has been primarily developed for high-grade gliomas such as glioblastoma and anaplastic astrocytoma, with additional investigation in other solid tumors and lymphomas. Vocimagene amiretrorepvec was originally developed by Tocagen and later by Denovo Biopharma, with clinical trials reaching up to phase II/III for brain cancers[1][4][5][7].
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