Precision BioSciences, Inc., an advanced gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, announced that the Company submitted Clinical Trial Applications (CTA) to initiate a phase 1 study evaluating PBGENE-HBV. PBGENE-HBV is the Company’s wholly owned in vivo gene editing programme designed to potentially cure chronic hepatitis B virus (HBV) by eliminating cccDNA, the key source of replicating hepatitis B virus, while also inactivating integrated HBV DNA in hepatocytes.
“The CTA submissions for PBGENE-HBV are important milestones for Precision as we pioneer this potentially curative therapy for chronic hepatitis B. These regulatory submissions are the culmination of our team’s dedication, commitment and highly productive interactions with global regulators as we develop the first clinical stage in vivo gene editing program for chronic hepatitis B virus, recognized as one of the largest global public health problems by the World Health Organization with an estimated 300 million people afflicted globally,” said Michael Amoroso, chief executive officer of Precision BioSciences. “Our comprehensive regulatory package, supported by robust non-human primate (NHP) safety studies, the gold standard model for predicting safety in humans, and efficacy in numerous preclinical models of hepatitis B including in NHPs enables us to proceed with planned regulatory submissions in multiple markets around the world. We recently bolstered our clinical team expertise while building a world class hepatitis scientific advisory board to assist in guiding execution of our global phase 1 trial. 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.”
Dr. Murray Abramson, senior vice president, head of clinical development added: “Current standard of care treatment with nucleos(t)ide analogs only offers 1-3% of patients a chance to achieve a functional cure. With our recent regulatory submissions, we are on the cusp of initiating a global first-in-human study for our wholly owned PBGENE-HBV programme. PBGENE-HBV is specifically designed to provide a better chance for a functional cure for chronic hepatitis B by eliminating the root source of viral replication, known as cccDNA. This is the first approach to directly target and eliminate cccDNA. We are excited at the prospect of bringing this potentially curative therapy to patients living with hepatitis B. We look forward to initiating the phase 1 study soon and expect to report data in 2025. More information about the safety of our clinical candidate and phase 1 clinical trial will be communicated in November when we plan to share a robust overview of the programme and our execution plans.”
Precision is on track to submit additional regulatory applications as part of its global phase 1 regulatory strategy for PBGENE-HBV. The next update on the PBGENE-HBV program is expected to take place before the American Association for the Study of Liver Diseases (AASLD) Annual Meeting in November. Details about how to participate in the update will be provided in advance.
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 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. PBGENE-HBV is a highly specific, novel therapeutic approach to treating patients with chronic HBV infection. It is designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures.
Precision BioSciences, Inc. is an advanced 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.
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