Precision BioSciences, Inc. announced that the company will present preclinical data for its PBGENE-HBV clinical candidate at the European Association for the Study of the Liver Congress (EASL), highlighting the differentiated ability of ARCUS to make efficient, durable, and targeted elimination edits. The poster presentation highlights data in primary human hepatocytes demonstrating the high specificity and lack of detectable off-target editing for PBGENE-HBV at therapeutically relevant doses. The poster will also showcase non-human primate data demonstrating good tolerability of a multi-dosing approach for the treatment of chronic hepatitis B. The data to be presented highlights that PBGENE-HBV specifically cuts HBV DNA leading to elimination of cccDNA and inactivation of integrated HBV DNA without impacting any sites in the human genome, including no editing-associated translocations in HBV infected primary human hepatocytes.

In addition, PBGENE-HBV was well-tolerated in non-human primates across multiple dose administrations, with only minor and transient elevations in liver transaminases that are normalized within 2 weeks and non-adverse changes in blood parameters. Preclinical safety data supports the advancement of PBGENE-HBV to clinical trials as a potentially curative treatment for chronic 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 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?s designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures.