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GalNAc-HNF4α-saRNA
- Development stage
- Preclinical
- Lead developer
- Beth Israel Deaconess Medical Center
- Modality
- RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
GalNAc-HNF4α-saRNA is a GalNAc-conjugated small activating RNA (saRNA) designed to target the long noncoding antisense RNA HNF4α-AS1 to restore the expression of the HNF4α-P1 isoform [3074]. Hepatocyte Nuclear Factor 4 Alpha (HNF4α) is a master transcription factor in hepatocytes that regulates mature liver function, metabolic homeostasis, and hepatocyte differentiation [3074]. In conditions like alcohol-associated hepatitis (AH) and non-alcoholic fatty liver disease (NAFLD), oxidative stress and inflammation lead to dysregulation of HNF4α isoforms, specifically a loss of the mature P1 isoform [3074]. By targeting HNF4α-AS1, this saRNA upregulates HNF4α-P1 expression, restoring the P1-specific transcriptome and normal liver function [3074]. The GalNAc conjugation enables targeted delivery to hepatocytes via the asialoglycoprotein receptor (ASGPR).
- Other names
- GalNAc-HNF4A saRNAGalNAc-HNF-4A saRNAGalNAc-HNF 4A saRNAGalNAc-HNF4a-saRNAGalNAc-HNF-4a-saRNAGalNAc-HNF 4a-saRNAHNF4A saRNA conjugateHNF-4A saRNA conjugateHNF 4A saRNA conjugateHNF4α-saRNA
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