Precision BioSciences has announced the approval of its Clinical Trial Application (CTA) in Moldova for PBGENE-HBV, an in vivo gene editing therapy designed to treat chronic hepatitis B. This marks a significant step forward as PBGENE-HBV becomes the first in vivo gene editing program targeting chronic hepatitis B virus (HBV) to enter global clinical trials.
The Phase 1 clinical program is set to begin, with the company actively screening patients at its clinical site in Moldova. Michael Amoroso, CEO of Precision BioSciences, stated, "Our first CTA approval for PBGENE-HBV represents another significant milestone as we complete our transition to a clinical stage in vivo gene editing company."
Targeting the Root Cause of Chronic Hepatitis B
Chronic hepatitis B, affecting approximately 300 million people globally, is driven by the persistence of HBV covalently closed circular DNA (cccDNA) and the integration of HBV DNA into the host genome within liver cells. Current treatments, such as nucleos(t)ide analogs, offer viral suppression but rarely lead to a functional cure and require lifelong administration. PBGENE-HBV aims to address this unmet need by directly targeting and eliminating cccDNA, the root cause of the disease.
PBGENE-HBV leverages Precision BioSciences' ARCUS gene editing platform, delivering an ARCUS nuclease-encoding mRNA to the liver via lipid nanoparticles. Once expressed in HBV-infected hepatocytes, the ARCUS nuclease specifically cuts a highly conserved sequence in the hepatitis B viral genome. This precision cutting is designed to eliminate cccDNA and inactivate integrated HBV genomes, potentially leading to functional cures.
Clinical Trial Strategy and Next Steps
Precision BioSciences has submitted multiple global clinical trial applications and plans to submit additional CTAs and IND filings as part of its global Phase 1 regulatory strategy for PBGENE-HBV. The company will provide updates on regulatory approvals as they are received.
Dr. Murray Abramson, Senior Vice President, Head of Clinical Development of Precision BioSciences, highlighted Moldova's expertise in hepatitis B clinical trials and the high prevalence of the disease in the country. "PBGENE-HBV is the first and only clinical stage approach to directly target and eliminate cccDNA, giving it the potential to achieve a functional cure," he noted.
An update on the PBGENE-HBV program, including the final preclinical safety package, will be shared with investigators and investors on November 15th, ahead of the American Association for the Study of Liver Diseases (AASLD) Annual Meeting.
