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SGT-94 is a tumor-targeted, systemically administered gene therapy investigational agent composed of cationic liposomes encapsulating a plasmid DNA encoding RB94, a truncated form of the retinoblastoma (RB) tumor suppressor gene[1][3][5]. The nanocomplex utilizes an anti-transferrin receptor (TfR) single-chain antibody fragment for specific delivery of the plasmid to tumor cells overexpressing TfR, particularly in genitourinary cancers such as metastatic bladder cancer[1]. Upon intravenous infusion, SGT-94 delivers and expresses RB94 selectively in tumor tissue, where RB94 induces apoptosis and inhibits tumor cell growth through restoration or enhancement of tumor suppressor gene activity. Clinical evidence from a phase 1 study showed the drug is well-tolerated and demonstrated signs of clinical activity, including partial and complete remissions, with no dose-limiting toxicities observed at tested doses[1][3][5]. SGT-94 was primarily developed to exploit tumor-selective gene expression for cancer therapy.
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