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INXN-2001 is an investigational DNA-based in vivo gene therapy consisting of an adenoviral vector (Ad-RTS-hIL-12) engineered to express human interleukin-12 (IL-12). Developed by Alaunos Therapeutics (formerly Ziopharm Oncology) in collaboration with Precigen, the therapy utilizes the RheoSwitch Therapeutic System (RTS) to provide inducible control over gene expression. The system is activated by the oral small molecule ligand veledimex (INXN-1001), which allows for dose-dependent regulation of IL-12 production. IL-12 is a potent proinflammatory cytokine that stimulates T-cell and natural killer (NK) cell activity, enhances antigen presentation, and inhibits tumor angiogenesis. The combination of intratumoral INXN-2001 and oral veledimex was primarily evaluated in Phase I/II clinical trials for the treatment of unresectable Stage III or IV melanoma, though development was later discontinued as the sponsor pivoted to other therapeutic platforms.
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