The FDA granted a fast track designation to clinical stage gene editing company, Precision BioSciences, for its investigational gene therapy, PBGENE-HBV. This in vivo gene editing program was developed to cure chronic hepatitis B by eliminating cccDNA, the key source of replicating the HBV, and inactivating integrated HBV DNA in hepatocytes.1
“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,” Precision CEO Michael Amoroso, said in a statement.1
Precision is evaluating PBGENE-HBV in its ongoing, global phase 1 trial, ELIMINATE-B, with clinical sites in the US, Moldova, Hong Kong, New Zealand, and the UK.1
“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,” Amoroso said.1
Precision developed its novel and proprietary genome editing platform, ARCUS. This platform is being developed for use in DNA genome insertion, deletion, and repair. Specifically, PBGENE-HBV uses a lipid nanoparticle (LNP) delivery of ARCUS mRNA.2 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 eg viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering 2 ARCUS nucleases in a single AAV).1
Precision BioSciences gene editing candidate, PBGENE-HBV.
For those not familiar with gene editing, it “is the process of modify genes of living organisms to improve our understanding of gene function and develop ways to use it to treat genetic or acquired diseases.”3 Gene editing therapies are being studied for several diseases and chronic conditions including cancer, diabetes, AIDS, hemophilia, and others.3
Learn more: Applying a Novel Gene Editing Therapy for Chronic Hepatitis B Treatment
Two gene therapies, Casgevy and Lyfgeni, have been FDA approved for sickle cell disease.4 For those developing gene editing technologies, there is a great deal of promise.
“A number of genetic conditions or even infectious diseases like hepatitis B [have] the root cause within your DNA,” Cassandra Gorsuch, PhD, Chief Scientific Officer, Precision BioSciences, said in a previous interview with Contagion. “And most of our treatment options across the board treat symptoms of diseases but not the actual causes of diseases. The idea of the gene editing approach is to use different types of technologies to permanently alter the DNA sequence, which is the underlying cause of disease.”2
Precision anticipates communicating updates on the full low-dose cohort, including multiple dose administrations, and data from higher dose levels throughout 2025.
