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prexigebersen

Development stage
Phase 2
Lead developer
Bio-Path Holdings
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

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].

Other names
Liposomal Grb2 antisense oligonucleotidePrexigebersen-A
02

Targets

Growth factor receptor-bound protein 2 mRNA (GRB2 mRNA)

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