Precision BioSciences has announced the submission of Clinical Trial Applications (CTAs) to initiate a Phase 1 study of PBGENE-HBV, a novel in vivo gene editing therapy designed to potentially cure chronic hepatitis B virus (HBV). This pioneering approach targets the elimination of covalently closed circular DNA (cccDNA), the primary source of HBV replication, and inactivates integrated HBV DNA within liver cells (hepatocytes).
Chronic hepatitis B, a significant global health concern affecting an estimated 300 million people worldwide, currently lacks curative treatment options. Existing therapies, such as nucleos(t)ide analogs, offer viral suppression but rarely lead to a functional cure, requiring lifelong administration and only providing a 1-3% chance of achieving it.
PBGENE-HBV: A Novel Therapeutic Approach
PBGENE-HBV represents a highly specific therapeutic strategy designed to address the limitations of current HBV treatments. By directly targeting and eliminating cccDNA and inactivating integrated HBV DNA, PBGENE-HBV aims to provide a better chance of achieving a functional cure for chronic hepatitis B. The therapy leverages Precision BioSciences' ARCUS® genome editing platform, which is designed for precise and efficient gene editing in vivo.
Michael Amoroso, CEO of Precision BioSciences, stated, "The CTA submissions for PBGENE-HBV are important milestones for Precision as we pioneer this potentially curative therapy for chronic hepatitis B... Our goal is clear, to generate robust clinical data for the patients afflicted with hepatitis B who are counting on us to significantly increase their chance of achieving a functional cure."
Clinical Trial and Future Expectations
The Phase 1 trial will be the first-in-human study for PBGENE-HBV. Precision BioSciences anticipates initiating the global Phase 1 trial soon and expects to report initial data in 2025. Further details regarding the safety profile of the clinical candidate and the Phase 1 clinical trial design are expected to be shared in November, prior to the American Association for the Study of Liver Diseases (AASLD) Annual Meeting.
Dr. Murray Abramson, Senior Vice President, Head of Clinical Development, noted, "With our recent regulatory submissions, we are on the cusp of initiating a global first-in-human study for our wholly owned PBGENE-HBV program... We are excited at the prospect of bringing this potentially curative therapy to patients living with hepatitis B."
Addressing Unmet Needs in HBV Treatment
Hepatitis B remains a leading cause of morbidity and mortality worldwide. An estimated 15% to 40% of individuals with chronic HBV infection may develop severe complications, including cirrhosis, liver failure, and hepatocellular carcinoma, underscoring the urgent need for more effective treatment strategies. PBGENE-HBV offers a potential paradigm shift in HBV management by targeting the root cause of the disease and aiming for a functional cure.
