Precision BioSciences gets FDA clearance for ELIMINATE-B trial of PBGENE-HBV

Precision BioSciences has secured a major regulatory breakthrough as the U.S. Food and Drug Administration (FDA) granted clearance for its Investigational New Drug (IND) application for PBGENE-HBV therapy. This milestone paves the way for the expansion of the company’s ELIMINATE-B trial, bringing its first-in-class gene editing treatment for chronic hepatitis B to clinical sites in the United States.

This development marks a significant step in the fight against hepatitis B, a chronic viral infection that affects millions globally, causing severe liver complications and an increased risk of liver cancer. Despite existing antiviral therapies, a true cure has remained elusive. Now, with the approval of PBGENE-HBV for clinical trials in the US, Precision BioSciences is positioning itself at the forefront of gene therapy innovations aimed at eradicating the root cause of chronic hepatitis B rather than merely suppressing its symptoms.

What Makes PBGENE-HBV Therapy Different from Traditional Hepatitis B Treatments?

Unlike conventional antiviral treatments, which primarily work by reducing viral load and preventing disease progression, PBGENE-HBV therapy takes a fundamentally different approach by using gene editing to eliminate cccDNA, the persistent viral genetic material that enables hepatitis B to replicate inside liver cells.

The therapy is built on Precision BioSciences’ proprietary ARCUS® gene editing platform, a technology designed to make precise genetic modifications with high specificity. By directly targeting cccDNA and inactivating integrated hepatitis B virus (HBV) DNA, the therapy aims to achieve what current treatments cannot—complete viral eradication.

While existing antiviral therapies such as nucleos(t)ide analogs can successfully suppress the virus and limit its damage, they do not eliminate cccDNA, meaning patients require lifelong treatment. Precision BioSciences’ approach, if proven effective, could provide a one-time or limited-course treatment that results in a functional cure for chronic hepatitis B.

How Will the ELIMINATE-B Trial Expand Following FDA Approval?

With FDA clearance secured, Precision BioSciences is expanding the ELIMINATE-B trial to include sites in the United States, joining existing clinical locations in Moldova, Hong Kong, and New Zealand. Massachusetts General Hospital’s Liver Center is set to be one of the first US locations to begin patient enrollment.

The trial is designed to assess PBGENE-HBV therapy in multiple cohorts, starting with low-dose administration and gradually increasing dosage levels to determine the optimal balance of safety and efficacy. Early results from the first cohort have already demonstrated encouraging safety and antiviral activity, prompting the company to move forward with higher dose levels to maximize therapeutic impact.

According to Murray A. Abramson, Head of Clinical Development at Precision BioSciences, expanding the trial into the US is a crucial step toward accelerating patient recruitment and increasing genetic diversity in the study population. The company has also indicated plans to extend the study to the UK later this year, reinforcing its commitment to a globally coordinated development strategy.

Why Is Chronic Hepatitis B Still a Global Health Challenge?

Chronic hepatitis B remains one of the most persistent and deadly viral infections worldwide. Despite widespread vaccination programs and improved access to antiviral therapies, the World Health Organization estimates that over 300 million people globally are living with the disease, with 1 to 2 million in the US alone.

Hepatitis B is particularly concerning due to its long-term health consequences. Many patients remain asymptomatic for years while the virus continues to damage their liver, eventually leading to cirrhosis, liver failure, or hepatocellular carcinoma (liver cancer). Studies suggest that up to 40% of individuals with chronic hepatitis B will develop serious liver complications over time.

The persistence of cccDNA in infected liver cells has been the biggest barrier to a cure, as the virus can reactivate even after long periods of suppression. Current treatments reduce viral replication but do not eliminate the underlying cccDNA, necessitating lifelong therapy to maintain control over the infection. This is why PBGENE-HBV therapy represents a potential game-changer—it directly targets and removes the source of chronic infection.

What Does This Mean for the Future of Gene Editing in Infectious Diseases?

Gene editing technologies have primarily been associated with genetic disorders, but the successful application of these techniques to infectious diseases could redefine treatment paradigms. Precision BioSciences’ work on PBGENE-HBV therapy demonstrates how gene editing can be leveraged to fight persistent viral infections in ways previously thought impossible.

If PBGENE-HBV therapy proves successful in clinical trials, it could open the door for similar gene editing approaches targeting other chronic viral diseases, such as HIV and herpes viruses, both of which persist in human cells in a manner similar to hepatitis B.

Michael Amoroso, President and CEO of Precision BioSciences, emphasized that the FDA clearance is a validation of the company’s approach to gene therapy, following multiple regulatory approvals in recent months. He noted that the progress of the ELIMINATE-B trial will be closely watched throughout 2025, with ongoing data updates expected as the trial progresses.

How Could This Impact Precision BioSciences and Investors?

For Precision BioSciences, securing FDA clearance for its leading gene editing candidate represents a major milestone in its pipeline development. The company, which is publicly traded on Nasdaq under the ticker DTIL, is likely to attract significant attention from investors, especially as PBGENE-HBV therapy advances further in clinical trials.

The potential for a functional cure for chronic hepatitis B presents an opportunity for substantial market disruption, particularly given the limitations of current therapies. If successful, the therapy could challenge the dominance of existing antiviral treatments, reshaping the standard of care for millions of patients worldwide.

Industry analysts have highlighted that Precision BioSciences’ strategic focus on gene editing for infectious diseases places it in a unique position within the biotech sector. While competitors have largely concentrated on genetic disorders, the company’s emphasis on viral infections could set a precedent for future advancements in the field.

As the company continues to advance its ELIMINATE-B trial, the biotech industry will be closely monitoring how gene editing fares as a viable therapeutic solution for chronic hepatitis B. With patient enrollment set to begin in the US, the next phase of the trial will provide critical insights into the long-term potential of this breakthrough approach.