- Approval in Hong Kong marks the first clinical trial application clearance of an in vivo gene editing approach for chronic hepatitis B in Hong Kong and the second CTA approval for PBGENE-HBV in 2024
- ELIMINATE-B is a global, multi-site study now actively recruiting patients; expected to report clinical data as it matures throughout 2025
- U.S. investigational new drug (IND) anticipated in 2025
DURHAM, N.C.--(BUSINESS WIRE)--Dec. 18, 2024-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, today announced that it has received Clinical Trial Application (CTA) approval in Hong Kong to study PBGENE-HBV in the ongoing ELIMINATE-B Phase I trial. PBGENE-HBV is Precision’s lead wholly owned in vivo gene editing program designed to cure chronic hepatitis B by eliminating cccDNA, the key source of replicating hepatitis B virus (HBV), and inactivating integrated HBV DNA in hepatocytes. The company is actively recruiting patients for the ELIMINATE-B trial in Moldova and has begun activating a top infectious disease clinical site in Hong Kong as part of its global regulatory and clinical operations strategy.
“We are pleased to receive our second CTA approval for PBGENE-HBV, expanding our ELIMINATE-B trial into a world-class HBV clinical trial site in Hong Kong,” said Dr. Murray Abramson, Senior Vice President, Head of Clinical Development of Precision BioSciences. “PBGENE-HBV has been designed to target a viral site that is prevalent across all HBV genotypes, including in Asia, and we believe that this will enable us to evaluate and provide access to as many patients as possible. In addition to Hong Kong, our clinical trial site in Moldova continues to execute as planned, and we look forward to sharing clinical data as it matures throughout 2025.”
“Over 400,000 individuals in Hong Kong are currently living with chronic hepatitis B, and many patients continue to develop liver cancer or cirrhosis even on existing long-term treatment with standard of care treatments,” said Dr. MF Yuen, DSc, MD, PhD, Chair Professor of Gastroenterology and Hepatology, Li Shu Fan Medical Foundation and Professor in Medicine, The University of Hong Kong. “By targeting and eliminating covalently closed circular (ccc) DNA, the root cause of the disease, PBGENE-HBV has the potential to completely eliminate the source of viral replication from the body, not just reduce it, which could transform the treatment landscape for patients in Hong Kong and millions of others worldwide. Our field has long searched for a novel way to eliminate the root cause of chronic hepatitis B, the cccDNA, and I look forward to further investigating PBGENE-HBV in clinic.”
Through its precision cutting, compact design, and simple structure, PBGENE-HBV is engineered to target the HBV viral genome and drive functional cures for patients with chronic hepatitis B. PBGENE-HBV leverages the ARCUS® gene editing platform by delivering an ARCUS nuclease-encoding mRNA to the liver via lipid nanoparticles. The ARCUS nuclease specifically cuts a highly conserved sequence in the hepatitis B viral genome and is designed to eliminate the root cause of the disease, cccDNA, and inactivate integrated HBV genomes. The ARCUS platform is derived from a naturally occurring enzyme and has been optimized for over 20 years into a ground-breaking and highly precise gene editing tool.
Precision has submitted multiple global clinical trial applications and remains on track for a U.S. IND in 2025 as part of its global Phase 1 regulatory strategy for PBGENE-HBV. The company will provide updates as it receives additional regulatory approvals to begin treating patients in those markets.
About Hepatitis B
Hepatitis B is a leading cause of morbidity in the US and death globally, with no curative options currently available for patients. In 2019, despite the availability of approved antiviral therapies, an estimated 300 million people globally and more than 1 million people in the US were estimated to have chronic hepatitis B infection. An estimated 15% to 40% of patients with HBV infections may develop complications, such as cirrhosis, liver failure, or liver cancer (hepatocellular carcinoma), which account for the majority of HBV-related deaths.
Chronic hepatitis B infection is primarily driven by persistence of HBV cccDNA and integration of HBV DNA into the human genome in liver cells, the primary source of hepatitis B surface antigen (HBsAg) in late-stage disease. Current treatments for patients with HBV infection include agents that result in long-term viral suppression as indicated by reduction of circulating HBV DNA, but these therapies do not eradicate HBV cccDNA, rarely lead to functional cure, and require lifelong administration.
About PBGENE-HBV
PBGENE-HBV is a potentially curative approach to treating patients with chronic HBV infection through a highly specific, novel therapeutic approach. PBGENE leverages the ARCUS® platform and is designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
The ARCUS® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV).