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EZN-2968 is a third-generation antisense oligonucleotide designed to inhibit the expression of hypoxia-inducible factor 1 alpha (HIF-1α), a transcription factor involved in tumor adaptation to hypoxia and upregulation of genes necessary for cancer cell survival. By specifically binding to HIF-1α mRNA, EZN-2968 blocks its translation and reduces HIF-1α protein levels, leading to inhibition of angiogenesis, tumor cell proliferation, and induction of apoptosis. Preclinical studies demonstrated potent down-regulation of HIF-1α and its downstream targets in various cancer models. Clinically, EZN-2968 has been evaluated in phase I trials for advanced solid tumors including liver cancer and lymphoma[2][4][6][7]. The drug was originally developed by Santaris Pharma and further developed by Enzon Pharmaceuticals.
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