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ANT2 GalNAc ASO
- 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
- Subcutaneous
01
Overview
ANT2 GalNAc ASO is a liver-targeted antisense oligonucleotide (ASO) designed to suppress the expression of mitochondrial ADP/ATP carrier 2 (ANT2) in hepatocytes. Developed through a collaboration involving Ionis Pharmaceuticals and Yale University researchers, the drug utilizes N-acetylgalactosamine (GalNAc) conjugation to ensure specific delivery to the liver via the asialoglycoprotein receptor. Suppression of hepatic ANT2 has been shown to rewire mitochondrial metabolism by inducing the expression of uncoupling protein 3 (UCP3), which in turn enhances hepatic fat oxidation and mitochondrial gluconeogenesis without depleting ATP or altering whole-body energy expenditure. This mechanism represents a novel approach for treating metabolic disorders such as metabolic dysfunction-associated steatotic liver disease (MASLD) and type 2 diabetes (T2D).
- Other names
- liver-targeted ANT2 GalNAc ASO
02
Targets
Adenine nucleotide translocator 2 (SLC25A5) mRNAASGPR (Asialoglycoprotein Receptor 1)
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