FDA Grants Fast Track to Precision BioSciences' PBGENE-HBV for Chronic Hepatitis B

Precision BioSciences, Inc., a pioneering entity in the realm of gene editing, has recently received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its innovative PBGENE-HBV program. This significant milestone underscores the urgent need for enhanced treatment options for individuals grappling with chronic hepatitis B. Utilizing its proprietary ARCUS® platform, Precision BioSciences aims to develop in vivo gene editing therapies targeting diseases with substantial unmet needs.

The primary focus of the PBGENE-HBV program is to provide a potential cure for chronic hepatitis B by tackling the root causes of the disease. It aims to eliminate covalently closed circular DNA (cccDNA), a critical source of the replicating hepatitis B virus, and deactivate integrated hepatitis B virus DNA within hepatocytes. This strategic approach could lead to groundbreaking advancements in the treatment of this widespread and challenging condition.

Michael Amoroso, President and CEO of Precision BioSciences, expressed satisfaction with the FDA's recognition of the program. He emphasized that the Fast Track designation is a testament to the pressing need for better treatment alternatives for those suffering from chronic hepatitis B. Amoroso highlighted the encouraging early safety and antiviral activity observed in the ongoing ELIMINATE-B trial, and shared his optimism about future collaborations with the FDA as the program progresses through clinical development.

The PBGENE-HBV program is currently being evaluated in the global Phase 1 ELIMINATE-B trial, which is being conducted in multiple countries including the United States, Moldova, Hong Kong, New Zealand, and the United Kingdom. Precision BioSciences is hopeful about sharing updates on the full low-dose cohort and data from higher dose levels throughout 2025, marking a year of critical developments for the program.

The Fast Track designation by the FDA is designed to facilitate the development and expedite the review process for drugs intended to address serious or life-threatening conditions with unmet medical needs. This status allows for more frequent communication and meetings with the FDA, potentially leading to accelerated timelines for regulatory approval. Additionally, it opens the door for Priority Review if certain criteria are met, further highlighting the significance of this designation for PBGENE-HBV.

Hepatitis B continues to be a leading cause of morbidity and mortality globally, with approximately 300 million people worldwide, and 1-2 million in the United States, living with chronic hepatitis B infection. Despite existing antiviral therapies, current treatment options primarily focus on long-term viral suppression and do not offer a curative solution. The persistence of HBV cccDNA and the integration of HBV DNA into the human genome in liver cells drive chronic hepatitis B, making effective eradication challenging.

Precision BioSciences' approach, through the use of the ARCUS® platform, represents a potential paradigm shift in treating chronic hepatitis B. The ARCUS® technology is distinct from other gene editing technologies, offering unique capabilities that enable precise and efficient therapeutic outcomes. By leveraging ARCUS®, the company is committed to developing gene editing candidates aimed at curing a broad spectrum of genetic and infectious diseases where current treatments fall short.

In summary, Precision BioSciences' PBGENE-HBV program, with its recent FDA Fast Track designation, marks a significant step forward in the quest to find a cure for chronic hepatitis B. The company's dedication to innovation and collaboration promises hope for millions of patients worldwide who continue to battle this challenging disease.