Precision BioSciences (NASDAQ: DTIL) has achieved a significant milestone with the first Clinical Trial Application (CTA) approval in Moldova for PBGENE-HBV, marking the initiation of the first in vivo gene editing program targeting chronic hepatitis B virus (HBV) in global clinical trials. This Phase 1 study aims to assess the safety and efficacy of PBGENE-HBV in patients with chronic HBV infection, a disease affecting nearly 300 million people worldwide. The company is actively pursuing additional CTA and IND approvals globally to broaden trial enrollment and expedite the development of this potential curative treatment.
Targeting the Root Cause of Chronic Hepatitis B
PBGENE-HBV is designed to address the root cause of chronic hepatitis B by eliminating covalently closed circular DNA (cccDNA) and inactivating integrated HBV DNA within hepatocytes. Current treatments often suppress the virus but rarely lead to a functional cure, with only a 1-3% chance of achieving this outcome. PBGENE-HBV leverages Precision BioSciences' proprietary ARCUS® platform, delivering mRNA encoding an ARCUS nuclease to the liver via lipid nanoparticles. This nuclease specifically targets and cuts a highly conserved sequence in the HBV viral genome, aiming to eradicate cccDNA and inactivate integrated HBV genomes.
Clinical Development and Regulatory Strategy
"At Precision, we remain laser-focused on execution, and 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," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "With rapid regulatory approval in hand, we are working diligently to screen and dose patients at our first clinical site in Moldova."
The choice of Moldova for the initial trial site is strategic, given the country's high prevalence of chronic hepatitis B and experience with Phase 1 clinical trials in this disease area. Precision BioSciences has assembled a clinical team with deep expertise in hepatitis B, guided by a scientific advisory board, to advance PBGENE-HBV towards dosing the first patient. The company has submitted multiple global clinical trial applications and plans to provide an update on the PBGENE-HBV program on November 15th, prior to the American Association for the Study of Liver Diseases (AASLD) Annual Meeting.
The Unmet Need in Hepatitis B Treatment
Hepatitis B remains a significant global health challenge, with an estimated 300 million people worldwide living with chronic infection. The disease can lead to severe complications, including cirrhosis, liver failure, and hepatocellular carcinoma, accounting for a substantial number of HBV-related deaths. Current treatments primarily focus on long-term viral suppression, but they do not eradicate cccDNA, the key source of viral replication. PBGENE-HBV represents a novel approach with the potential to achieve functional cures by directly targeting and eliminating cccDNA.
ARCUS® Platform: A Novel Gene Editing Approach
The ARCUS® platform is a distinctive genome editing technology that differs from other approaches in its cutting mechanism, compact size, and simplified structure. These features may enable ARCUS nucleases to drive more precise and predictable therapeutic outcomes. The platform is being utilized to develop in vivo gene editing therapies for various sophisticated gene edits, including gene insertion, elimination, and excision.
Forward-Looking Statements
This news release contains forward-looking statements that involve risks and uncertainties. The company's actual results may differ materially from those expressed or implied in these statements due to various factors, including the clinical development and expected safety, efficacy, and benefit of PBGENE-HBV, the timing of regulatory processes, and the success of preclinical and clinical studies.
