Precision BioSciences Receives FDA Fast Track Designation for PBGENE-HBV, an In Vivo Gene Editing Therapy for Chronic Hepatitis B

DURHAM, N.C.–(BUSINESS WIRE)–April 15, 2025– Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical-stage gene editing company utilizing its proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for PBGENE-HBV. This is the Company’s lead wholly owned in vivo gene editing program designed to cure chronic hepatitis B by eliminating covalently closed circular DNA (cccDNA), the key source of replicating hepatitis B virus (HBV), and inactivating integrated HBV DNA in hepatocytes (liver cells).

“We are pleased to receive Fast Track designation from the FDA for PBGENE-HBV and believe this classification underscores the urgent need for improved treatment options for patients living with chronic hepatitis B,” said Michael Amoroso, President and Chief Executive Officer at Precision BioSciences. “We’ve been encouraged by the initial safety and antiviral activity we have observed in the ELIMINATE-B trial and look forward to continuing to work closely with the FDA as we progress PBGENE-HBV through clinical development.”

Precision is currently evaluating PBGENE-HBV in the ongoing global Phase 1 ELIMINATE-B trial, with clinical investigation in the United States, Moldova, Hong Kong, New Zealand, and the United Kingdom. The company anticipates sharing updates on the full low-dose cohort, including multiple dose administrations, and data from higher dose levels throughout 2025.

Fast Track designation is intended to facilitate the development and expedite the review of drugs that are intended to treat serious or life-threatening conditions and address an unmet medical need. This designation allows for more frequent meetings and communications with the FDA and the potential for rolling review of marketing applications, as well as Priority Review if relevant criteria are met.

About PBGENE-HBV (Viral Elimination Program):

PBGENE-HBV is Precision’s wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, designed with the potential to cure chronic hepatitis B infection. Affecting an estimated 300 million people worldwide, chronic hepatitis B currently has no curative options. PBGENE-HBV comprises a meganuclease-encoding mRNA encapsulated in a lipid nanoparticle (LNP) and is designed to eliminate cccDNA and inactivate integrated HBV DNA; these are the persistent forms of the virus that serve as templates for viral replication and antigen production. By targeting these, PBGENE-HBV is designed to be curative by addressing the root cause of chronic HBV infection.

About Hepatitis B:

Hepatitis B is a major cause of illness and death globally, with no current cure. Despite available antiviral therapies that suppress the virus, an estimated 300 million people worldwide and 1-2 million in the US live with chronic HBV infection. A significant percentage of these individuals may develop severe complications like cirrhosis, liver failure, or liver cancer, which are the primary causes of HBV-related deaths. The persistence of HBV cccDNA and the integration of HBV DNA into the host genome in liver cells drive chronic infection and the production of hepatitis B surface antigen (HBsAg). Existing treatments offer long-term viral suppression but do not eliminate cccDNA, rarely lead to a functional cure, and require lifelong administration.