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GalNAc-Mdm2ASO
- 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
GalNAc-Mdm2ASO is a liver-targeted antisense oligonucleotide (ASO) therapy designed to treat metabolic-dysfunction-associated steatotic liver disease (MASLD). The molecule consists of an antisense sequence targeting MDM2 (Mouse double minute 2 homolog) mRNA, conjugated to a triantennary N-acetylgalactosamine (GalNAc) moiety. This conjugation enables specific delivery to hepatocytes by binding to the asialoglycoprotein receptor (ASGPR). By reducing the hepatic expression of MDM2, an E3 ubiquitin ligase that is upregulated in MASLD, the drug mitigates liver steatosis, inflammation, and fibrosis. Preclinical studies in rodent models have demonstrated its ability to improve liver injury, reduce the accumulation of hepatic lipids (cholesterol, diacylglycerol, and ceramide), and alleviate obesity-related insulin resistance and hyperlipidemia.
- Other names
- GalNAc-conjugated MDM2 antisense oligonucleotidehepatocyte-specific triantennary N-acetylgalactosamine-conjugated antisense oligonucleotide targeting MDM2GalNAc-MDM2 ASOGalNAc-MDM-2 ASOGalNAc-MDM 2 ASO
02
Targets
MDM2 (Mouse double minute 2 homolog)ASGPR (Asialoglycoprotein Receptor 1)
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