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MARC1 GalNAc-siRNA
- Development stage
- Unknown
- Lead developer
- AstraZeneca
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
MARC1 GalNAc-siRNA refers to a class of RNA interference (RNAi) therapeutics designed to target and silence the mitochondrial amidoxime-reducing component 1 (MARC1, also known as MTARC1) gene in hepatocytes. These therapeutics utilize N-acetylgalactosamine (GalNAc) conjugation to facilitate targeted delivery to the liver via the asialoglycoprotein receptor (ASGPR). Silencing MARC1 is a genetically validated strategy for the treatment of metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH), as loss-of-function variants in the MARC1 gene (such as rs2642438) have been shown to protect against hepatic steatosis, liver cirrhosis, and hepatocellular carcinoma. Multiple companies, including AstraZeneca (in collaboration with Silence Therapeutics), Novo Nordisk (with NNC0581-0001), and OliX Pharmaceuticals (with OLX702A, licensed to Eli Lilly), are developing GalNAc-siRNA candidates targeting MARC1.
- Other names
- MTARC1 GalNAc-siRNAMTARC-1 GalNAc-siRNAMTARC 1 GalNAc-siRNAMTARC1 siRNAMTARC-1 siRNAMTARC 1 siRNAmARC1 GalNAc-siRNAmARC-1 GalNAc-siRNAmARC 1 GalNAc-siRNAmARC1 siRNAmARC-1 siRNAmARC 1 siRNA
02
Targets
MTARC2 (Mitochondrial amidoxime reducing component 2)MTARC1 (Mitochondrial amidoxime-reducing component 1)
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