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REGEN001
- Development stage
- Preclinical
- Lead developer
- Apterna Therapeutics
- 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
REGEN001 is a preclinical liver-targeted RNA therapeutic being developed by Apterna Therapeutics. It consists of a small interfering RNA (siRNA) targeting CCAAT/enhancer-binding protein beta (C/EBPβ, or CEBPB) conjugated to N-acetylgalactosamine (GalNAc) for hepatocyte-specific delivery via the asialoglycoprotein receptor (ASGPR). C/EBPβ is a transcription factor that acts as a master regulator of hepatic metabolism, inflammation, and fibrosis. By silencing C/EBPβ expression in hepatocytes, REGEN001 aims to restore core liver function and promote hepatic resilience. The therapeutic is initially being developed to restore liver reserve in liver cancer patients who are currently ineligible for curative surgery due to poor liver function, with planned future expansions into advanced liver dysfunction (cirrhosis and transplant avoidance) and metabolic liver diseases (fatty liver disease and obesity-related liver dysfunction).
- Other names
- GalNAc-siCEBPBGalNAc-siCEBPβ
02
Targets
CCAAT/enhancer-binding protein beta mRNAASGPR (Asialoglycoprotein Receptor 1)
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