Drug intelligence / Profile preview

EPI-003

Development stage
Phase 1
Lead developer
Epigenic Therapeutics
Modality
Small Molecules, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

EPI-003 is a first-in-class antiviral gene therapy developed for the treatment of chronic hepatitis B virus (HBV) infection. It is administered via intravenous infusion and specifically targets the liver using epigenetic modification mechanisms. The drug consists of mRNA encoding an epigenetic modulation protein and a guide RNA targeting HBV-specific genes, encapsulated in a lipid nanoparticle (LNP) delivery system. EPI-003 is designed to induce precise epigenetic modifications on the HBV genome, directly blocking transcription from covalently closed circular DNA (cccDNA) and integrated viral DNA without altering the underlying gene sequence. This approach aims to sustainably reduce HBV surface antigen (HBsAg) and viral DNA levels, potentially offering a functional or complete cure for chronic hepatitis B patients. Preclinical studies have shown promising results with significant reduction in viral markers without rebound[1][2][3][4]. Developers: Epigenic Therapeutics

02

Targets

Integrated HBV DNA (Integrated hepatitis B virus DNA)

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