Milestone Regulatory Clearance for Gene-Editing Therapy

On March 19, 2025, Precision BioSciences announced that the FDA has cleared its Investigational New Drug (IND) application for PBGENE-HBV, marking the first-ever in vivo gene-editing therapy trial for chronic hepatitis B virus (HBV) infection in the United States. PBGENE-HBV is a meganuclease-edited, lipid nanoparticle-delivered therapy designed to disrupt HBV DNA and permanently reduce hepatitis B surface antigen (HBsAg) levels.

Expansion of the Global ELIMINATE-B Clinical Program

With the IND clearance, Precision will expand its ongoing ELIMINATE-B Phase 1 trial—already enrolling in Moldova, Hong Kong, and New Zealand—to include US patients. The protocol aims to define optimal dose and dosing schedules, with early data from international cohorts showing PBGENE-HBV is well-tolerated and exhibits substantial antiviral activity, even at lower dose levels.

Hope for a Functional Cure in Hepatitis B

This marks a pivotal moment for patients worldwide: current HBV therapies suppress viral replication but rarely lead to functional cure. PBGENE-HBV’s mechanism—targeting and inactivating covalently closed circular DNA (cccDNA)—holds promise to induce long-lasting remission and potentially cure chronic infection, a transformative outcome for millions living with HBV.

Outlook: Pioneering Gene Editing in Infectious Disease

Early safety and efficacy results from the ELIMINATE-B trial are anticipated later in 2025. Industry observers view this FDA milestone as a validation for in vivo gene-editing strategies in high-burden infectious diseases and as proof of concept for future gene therapies beyond HBV.