Precision BioSciences' PBGENE-HBV IND Approved by FDA to Target Chronic Hepatitis B

Precision BioSciences, Inc., a company at the forefront of gene editing technology, has announced a significant breakthrough for its lead program, PBGENE-HBV. The U.S. Food and Drug Administration (FDA) has granted clearance for the Investigational New Drug (IND) application for this innovative therapy, marking it as the first in vivo gene editing treatment to enter clinical trials in the United States for chronic hepatitis B. PBGENE-HBV is designed to address the core issues of chronic hepatitis B by targeting and eliminating covalently closed circular DNA (cccDNA) and inactivating integrated hepatitis B virus (HBV) DNA within liver cells.

The approval of the IND application is a crucial step in the progress of PBGENE-HBV, enabling the expansion of Phase 1 of the ELIMINATE-B study into the U.S. Previously, this study was already underway in Moldova, Hong Kong, and New Zealand. Now, U.S. patients can participate in the trial, which will start at the Liver Center at Massachusetts General Hospital, adding a significant location to the study's roster.

Michael Amoroso, the President and CEO of Precision BioSciences, emphasized the importance of this advancement. He highlighted how this clearance represents a pivotal moment for the gene editing field, as it brings potentially curative treatments to major markets burdened by chronic diseases. With this achievement, the company has demonstrated the robustness of its global development strategy and its commitment to addressing high unmet medical needs.

The ELIMINATE-B trial has already shown encouraging safety and efficacy outcomes following the initial administration of PBGENE-HBV at its lowest dosage. The trial is proceeding with the administration of additional doses to patients in the first cohort and is preparing to escalate to a higher dose in the second cohort. This strategy aims to identify the optimal dosage that effectively eliminates cccDNA and inactivates integrated HBV DNA while ensuring patient safety. There are also plans to expand the study to the United Kingdom, which will further bolster patient recruitment and diversity within the trial.

Dr. Murray A. Abramson, head of Clinical Development at Precision BioSciences, expressed excitement about the trial's extension to the U.S., underscoring the collaborative efforts with investigators in the other participating countries. The goal of the trial remains to advance potential curative treatments for hepatitis B, a disease that currently affects millions globally and lacks definitive curative therapies.

Hepatitis B significantly impacts individuals worldwide, causing various health complications such as cirrhosis, liver failure, and liver cancer. Despite existing antiviral therapies, these treatments only suppress the virus and do not eradicate it, leading to lifelong medication for patients. Precision BioSciences aims to change this paradigm with PBGENE-HBV, providing a path towards a functional cure for those affected.

Precision BioSciences is leveraging its proprietary ARCUS® platform for this endeavor. ARCUS is distinct in its smaller size and simpler structure, leading to more precise and intended therapeutic outcomes. The platform is designed for sophisticated gene edits, including the elimination of viral DNA, making it a promising tool for developing lasting cures for various genetic and infectious diseases where current treatments fall short.

In summary, PBGENE-HBV represents a pioneering effort in the realm of gene editing therapies for chronic hepatitis B. With ongoing trials and a strategic global approach, Precision BioSciences is moving forward in its mission to provide groundbreaking treatment options for those affected by this challenging disease.