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Prexigebersen is a neutral-charge, liposome-incorporated antisense oligonucleotide drug designed to inhibit the synthesis of growth factor receptor-bound protein 2 (Grb2), an adaptor protein involved in cancer progression via the RAS activation pathway. By binding to Grb2 mRNA, prexigebersen blocks its translation and reduces Grb2 protein levels, thereby inhibiting cell proliferation in cancers that overexpress this target. The drug is being developed primarily for hematologic malignancies such as acute myeloid leukemia (AML) and chronic myeloid leukemia (CML), with additional investigation in solid tumors and obesity-related indications. Prexigebersen has received orphan drug designation from both the FDA and EMA for AML and CML[1][4][5][6].
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