Precision BioSciences Reports Initial Safety and Antiviral Activity of PBGENE-HBV in ELIMINATE-B Trial

Precision BioSciences, Inc., a clinical-stage gene editing company, has announced promising initial results from the ELIMINATE-B trial, a significant study targeting chronic Hepatitis B. Utilizing the company's proprietary ARCUS® platform, the trial focuses on PBGENE-HBV, a treatment designed to combat the virus by targeting the root cause, covalently closed circular DNA (cccDNA).

The ELIMINATE-B study is structured to evaluate PBGENE-HBV across multiple ascending doses. The initial phase involved three patients in the lowest dose cohort, receiving a 0.2 mg/kg dose. These first results indicate that PBGENE-HBV was safe and well-tolerated, with no significant adverse events reported. This outcome is a crucial step as the company prepares to administer additional doses and explore higher dose levels.

PBGENE-HBV operates as an mRNA encoding ARCUS, encapsulated within a lipid nanoparticle (LNP). This design aims to safely deliver the gene editing technology to patients. The trial's protocol includes three administrations per dose level, aiming to enhance cumulative viral editing for maximum reduction of Hepatitis B surface antigen (HBsAg). Initial results showed a substantial decrease in HBsAg in two of the three patients, supporting the antiviral potential of this approach.

The primary objective of the ELIMINATE-B trial is to determine the optimal dosing for safely removing cccDNA and inactivating integrated Hepatitis B virus DNA. This trial marks the second clinical validation of ARCUS in vivo gene editing, following earlier successes in other applications. This progress demonstrates the potential of ARCUS technology in providing therapeutic solutions where conventional treatments have fallen short.

Key figures in the trial emphasized the importance of safety monitoring in these early stages. Murray A. Abramson, MD, MPH, Head of Clinical Development, highlighted the careful preclinical safety experiments and mRNA optimization efforts that were crucial in reaching this phase without safety concerns. The trial's design reflects a commitment to patient safety while exploring new frontiers in gene editing technology.

Principal Investigator Alina Jucov, MD, PhD, expressed enthusiasm about the trial's early outcomes. She noted the high interest from patients and the promising safety and activity profile of PBGENE-HBV. The trial is currently recruiting patients in Moldova, Hong Kong, and New Zealand, with plans to expand to the U.S. and U.K., reflecting its global scope and ambition.

Michael Amoroso, President and CEO of Precision BioSciences, highlighted the significance of the trial's early results for the company and the advancement of ARCUS technology. The progress in treating chronic Hepatitis B with gene editing represents a crucial step forward for the company and the field of gene therapy.

Cassie Gorsuch, PhD, Chief Scientific Officer, discussed the alignment between preclinical and clinical data, indicating that PBGENE-HBV is achieving what previous treatments have not—directly targeting the viral replication source in cccDNA and inactivating integrated disease.

Precision BioSciences continues to drive innovation in gene editing, with the ELIMINATE-B trial reflecting its commitment to tackling diseases with high unmet medical needs. As the study progresses, the company plans to share detailed clinical data throughout 2025, further contributing to the understanding and potential treatment of chronic Hepatitis B.