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Temferon is an investigational, ex-vivo genetically modified cell therapy developed for cancer treatment, primarily glioblastoma multiforme (GBM). It is based on autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that are engineered using a lentiviral vector to express human interferon-alpha 2 (IFN-α2), under the control of the Tie2 promoter and post-transcriptional miRNA elements. After engraftment, these cells differentiate into Tie2-expressing monocytes/macrophages (TEMs), which home to tumors and locally release IFN-α within the tumor microenvironment. This targeted delivery aims to reprogram the tumor microenvironment by restoring immune function and counteracting tumor growth while minimizing systemic toxicity associated with IFN-α therapies. Temferon has received orphan drug designation from the FDA for GBM with unmethylated MGMT promoter status and is in Phase 1/2 clinical trials for this indication. Developers: Genenta Science
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