Drug intelligence / Profile preview

PBGENE-HBV

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

Overview

PBGENE-HBV is an investigational gene editing therapy developed for the potential cure of chronic hepatitis B virus (HBV) infection. It consists of a meganuclease-encoding mRNA delivered via lipid nanoparticle (LNP), designed to eliminate covalently closed circular DNA (cccDNA) and inactivate integrated HBV DNA within hepatocytes—the persistent forms responsible for ongoing viral replication and antigen production. By targeting these reservoirs, PBGENE-HBV aims to achieve a functional cure, defined as sustained undetectable levels of HBV DNA and hepatitis B surface antigen (HBsAg) after treatment cessation. The therapy utilizes Precision BioSciences' proprietary Arcus genome editing platform and is currently being evaluated in the Phase 1 ELIMINATE-B clinical trial, with preclinical data demonstrating up to 99% viral eradication in animal models[1][3][4][5][6][7].

02

Targets

Integrated HBV DNA (Integrated hepatitis B virus DNA)cccDNA (Hepatitis B virus covalently closed circular DNA)

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