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hnf4α srRNA
- Development stage
- Phase 1
- Lead developer
- Shanghai ChangZheng Hospital
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
hnf4α srRNA is a small activating RNA (saRNA) therapeutic designed to upregulate the expression of hepatocyte nuclear factor 4 alpha (HNF4A), a master transcription factor critical for liver development and function. By increasing HNF4A levels, this drug aims to modulate gene expression programs involved in metabolism and differentiation of hepatic cells. It is being developed primarily for the treatment of metastatic colorectal carcinoma and intrahepatic cholangiocarcinoma, with early clinical trials initiated by Shanghai Changzheng Hospital. The mechanism involves direct modulation of HNF4A gene activity to restore or enhance its tumor-suppressive functions in cancerous tissues[1].
- Other names
- HNF4A saRNAHNF-4A saRNAHNF 4A saRNAHNF4A small activating RNAHNF-4A small activating RNAHNF 4A small activating RNAhepatocyte nuclear factor 4 alpha saRNA
02
Targets
HNF4A (Hepatocyte nuclear factor 4 alpha)
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