Precision BioSciences Advances PBGENE-HBV to Phase 1 Trial for Chronic Hepatitis B Treatment
Key Insights
Precision BioSciences received approval for its Clinical Trial Application for PBGENE-HBV, a gene editing therapy for chronic hepatitis B (search).
The Phase 1 clinical program for PBGENE-HBV has commenced in Moldova, with patient screening underway and clinical data expected in 2025.
PBGENE-HBV is designed to eliminate cccDNA and inactivate integrated HBV DNA (search), addressing the root cause of viral persistence in chronic hepatitis B (search).
Precision BioSciences, Inc. (Nasdaq: DTIL) has announced the advancement of its PBGENE-HBV program into a Phase 1 clinical trial for the treatment of chronic hepatitis B (search). The company received approval for its Clinical Trial Application (CTA) and has initiated the trial in Moldova, with patient screening currently in progress. Clinical data from the Phase 1 trial is expected in 2025.
PBGENE-HBV: A Novel Approach to Chronic Hepatitis B
PBGENE-HBV is a first-in-human in vivo gene editing program designed to potentially cure chronic hepatitis B (search) by eliminating covalently closed circular DNA (cccDNA) and inactivating integrated HBV DNA (search). This dual-targeting approach aims to address the root cause of viral persistence in chronic hepatitis B, which affects an estimated 300 million people worldwide.
Michael Amoroso, Chief Executive Officer at Precision BioSciences, stated, "PBGENE-HBV represents the very first clinical stage gene editing program for chronic hepatitis B (search) utilizing a differentiated dual modality targeting the elimination of cccDNA and inactivation of integrated HBV genomes."
Clinical Trial Details and Regulatory Strategy
The Phase 1 clinical trial for PBGENE-HBV is underway in Moldova, a country with experience in executing and enrolling patients in early- and mid-stage hepatitis B (search) clinical trials. Precision BioSciences has also submitted additional regulatory applications globally and is awaiting approval. Preclinical data supports the ability of PBGENE-HBV to specifically target and eliminate hepatitis B cccDNA and inactivate integrated HBV DNA (search) without off-target effects.
PBGENE-3243: Targeting m.3243-Associated Mitochondrial Disease
Precision BioSciences is also developing PBGENE-3243 (search), a potential treatment for m.3243-associated mitochondrial diseases. This program targets mutant mitochondrial DNA (search), allowing wild-type mitochondrial DNA to repopulate and improve cellular function. The company expects to submit an Investigational New Drug (IND) application or CTA for PBGENE-3243 in 2025. The m.3243-associated mitochondrial disease (search) affects approximately 20,000 people in the US alone.
ARCUS Platform and Gene Editing Capabilities
Precision BioSciences utilizes its proprietary ARCUS genome editing platform, which differs from other technologies in its cutting mechanism, smaller size, and simpler structure. ARCUS nucleases are designed to drive more intended and defined therapeutic outcomes through gene insertion, elimination, and excision.
Financial Update and Future Outlook
As of September 30, 2024, Precision BioSciences had approximately $121.3 million in cash, cash equivalents, and restricted cash. The company expects its existing cash and cash equivalents, along with potential near-term cash from CAR T transactions and operational receipts, to extend its cash runway into the second half of 2026. This is projected to be sufficient to propel two wholly owned programs through Phase 1 data readouts in 2025 and 2026.