Precision BioSciences, Inc. announced that the company has received pre-IND regulatory feedback from the U.S. Food and Drug Administration (FDA) and ex-U.S. agencies. Receipt of this regulatory feedback provides alignment and clarity on Precision's final IND/CTA-enabling preclinical plans and clinical strategy for PBGENE-HBV prior to advancement into Phase 1 clinical studies. The pre-IND meeting with FDA provided feedback on overall design for the proposed first-in-human clinical study as well as feedback on the toxicology and specificity assessments.

This interaction builds on the engagement and alignment achieved through early scientific advice during the FDA INTERACT meeting in July 2023. Similar, productive consultations have been held with regulators in ex-U.S. countries. Collectively, the feedback and overall alignment obtained from multiple regulatory authorities validates Precision?s path towards a planned IND and/or CTA filing for PBGENE-HBV in 2024.

Precision is committed to rapidly advancing PBGENE-HBV into a global clinical Phase 1 study and is currently expanding site selection efforts with specialized centers focused on the treatment of Chronic Hepatitis B in several countries. Simultaneously, manufacturing is proceeding to supply drug for all planned nonclinical and clinical activities. 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, resulting in degraded cccDNA and a reduction in HBsAg.