Applying a Novel Gene Editing Approach to Chronic Hepatitis B

This content was originally published on our sister site, Contagion Live.

“A number of genetic conditions or even infectious diseases like hepatitis B [have] the root cause within your DNA,” Cassandra Gorsuch, PhD, head of Gene Therapy Discovery, Precision BioSciences, said. "And most of our treatment options across the board treat symptoms of diseases but not the actual causes of diseases. The idea of gene editing approach is to use different types of technologies to permanently alter the DNA sequence, which is the underlying cause of disease.”

The clinical stage biotechnology company that has created a novel and proprietary genome editing platform, ARCUS. This platform is being developed for use in DNA genome insertion, deletion, and repair. Specifically, their PBGENE-HBV product gene editing candidate uses a lipid nanoparticle (LNP) delivery of ARCUS mRNA.

Data from Precision’s preclinical study demonstrated ARCUS targeted and degraded HBV covalently closed circular (cccDNA) by 85% and reduced expression of HBV’s Surface Antigen (HBsAg) by 77% in HBV-infected primary human hepatocytes (PHH). The cccDNA plays a central role in chronic HBV.

Similar levels of editing were achieved in novel mouse and NHP models following its LNP delivery resulting in a 96% reduction in HBsAg.

Although this is early in the development process, the company plans to move forward with PBGENE-HBV, and they will file for an FDA investigational new drug application in 2024.

Gorsuch spoke to Contagion about this gene editing candidate, how their platform differs from other technologies such as CRISPR/Cas9, and the company’s hopes for its potential place in the market for HBV treatment.