Precision BioSciences Unveils New Preclinical Safety Data for PBGENE-HBV at Liver Congress

Precision BioSciences, Inc., a leading gene editing company utilizing the proprietary ARCUS® platform, has announced the presentation of preclinical data for its therapeutic candidate, PBGENE-HBV, at the European Association for the Study of the Liver Congress (EASL). The presentation will underscore the unique ability of ARCUS to perform precise, enduring, and targeted genetic modifications. The data emphasizes the high specificity and absence of off-target editing in human hepatocytes treated with PBGENE-HBV at therapeutically relevant doses.

PBGENE-HBV has demonstrated promising results in preclinical trials, showing specific targeting and cutting of HBV DNA while entirely avoiding the human genome. This precision eliminates the covalently closed circular DNA (cccDNA) and inactivates integrated HBV DNA, which are crucial for the persistence of the virus in chronic hepatitis B infections. The data also highlights that in non-human primates, multiple doses of PBGENE-HBV were well-tolerated, with only minor and transient increases in liver enzymes that normalized within two weeks, and no adverse changes in blood parameters were observed.

Chronic hepatitis B is a significant health concern globally, affecting around 300 million people, with over 1 million cases in the United States alone. Current antiviral treatments manage to suppress the virus but do not eradicate cccDNA, leading to lifelong treatment requirements and seldom achieving a functional cure. PBGENE-HBV aims to address these gaps by offering a potential curative treatment through its precise gene-editing capabilities.

The ARCUS® platform, which distinguishes itself by its smaller size and simpler structure compared to other gene-editing technologies, enables PBGENE-HBV to efficiently eliminate and inactivate HBV DNA. This platform's key features include its ability to deliver specific and durable gene edits, which may lead to more defined and intended therapeutic outcomes. These capabilities set ARCUS apart in the field of gene editing, positioning it as a promising tool for developing lasting cures for a range of genetic and infectious diseases.

Jeff Smith, PhD, Chief Research Officer of Precision BioSciences, expressed confidence in the data presented, highlighting the potential of PBGENE-HBV as a curative treatment for chronic hepatitis B. He emphasized that the preclinical results validate the capabilities of the ARCUS® platform in delivering safe and effective in vivo gene editing therapies. The company is preparing to transition PBGENE-HBV to clinical trials, with plans to submit an Investigational New Drug Application (IND) and/or Clinical Trial Application (CTA) by 2024.

In summary, Precision BioSciences' presentation at the EASL Congress will highlight the potential of PBGENE-HBV as a novel therapeutic approach for chronic hepatitis B. The preclinical data demonstrates the gene-editing candidate's high specificity and safety, supporting its advancement to clinical trials. If successful, PBGENE-HBV could offer a finite and potentially curative treatment option for patients suffering from this chronic viral infection.