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GalNAc-siRNA targeting ALYREF
- Development stage
- Preclinical
- Lead developer
- The University of Hong Kong
- 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
GalNAc-siRNA targeting ALYREF is a preclinical-stage GalNAc-conjugated small interfering RNA (siRNA) designed to target and silence the expression of ALYREF (Aly/REF export factor) [1050]. ALYREF is an RNA export adaptor that mediates liquid-liquid phase separation (LLPS) and nuclear export of cell cycle-related mRNAs, driving oncogenic gene expression programs in hepatocellular carcinoma (HCC) [1050]. By conjugating the siRNA to N-acetylgalactosamine (GalNAc), the therapeutic is selectively delivered to hepatocytes via the asialoglycoprotein receptor (ASGPR). In preclinical mouse models of hepatocellular carcinoma, targeted delivery of GalNAc-siRNA against ALYREF significantly inhibited tumor growth, demonstrating therapeutic potential for HCC progression [1050].
- Other names
- GalNAc-conjugated siRNA targeting ALYREFGalNAc-siRNA against ALYREF
02
Targets
ALYREF (THO complex subunit 4)
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