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Inodiftagene vixteplasmid is a recombinant DNA plasmid gene therapy designed to selectively target and kill malignant cells. The plasmid encodes the diphtheria toxin A (DTA) gene under the control of the H19 promoter, which is highly active in cancer cells but not in normal tissue. When administered, typically via intravesical instillation for bladder cancer, it leads to expression of diphtheria toxin specifically within tumor cells that overexpress H19 RNA, resulting in targeted cell death. The drug was developed primarily for non-muscle-invasive bladder cancer unresponsive to BCG therapy and was also investigated for other solid tumors including pancreatic, ovarian, and liver cancers. Despite initial promising results showing tumor ablation and recurrence prevention in early trials, later phase 2 studies were discontinued due to insufficient efficacy[3][5][6].
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