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ME3 antisense oligonucleotide

Development stage
Preclinical
Lead developer
Ionis Pharmaceuticals
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
Administration
Intrathecal
01

Overview

ME3 antisense oligonucleotide is an experimental RNA-targeted therapy developed by Ionis Pharmaceuticals in collaboration with MD Anderson Cancer Center. It utilizes a collateral lethality approach to treat pancreatic cancer and other malignancies characterized by the homozygous deletion of the SMAD4 gene, which frequently results in the co-deletion of the neighboring mitochondrial malic enzyme 2 (ME2) gene. Since ME2 and ME3 provide redundant mitochondrial malic enzyme activity essential for NADPH production and ROS homeostasis, the ASO-mediated depletion of ME3 selectively induces apoptosis in ME2-null cancer cells while sparing normal ME2-intact cells. Preclinical studies have demonstrated that ME3 ASOs can significantly reduce tumor burden in pancreatic cancer models.

Other names
ME3 ASOME-3 ASOME 3 ASOanti-sense oligonucleotide to target ME3
02

Targets

Malic enzyme 3 (NADP(+)-dependent, mitochondrial) mRNA

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