Gene editing company Precision BioSciences (Nasdaq:DTIL) has announced that preclinical research involving in vivo gene editing targeting hepatitis B virus (HBV) has been published in Molecular Therapy.
In the research, the company used an engineered ARCUS nuclease (ARCUS-POL) targeting the Hepatitis B virus (HBV) genome.
In this preclinical study, ARCUS efficiently targeted and degraded HBV covalently closed circular DNA (cccDNA) by 85%.
“Today, chronic hepatitis B infection is a life-threatening condition managed by medicines that can suppress the virus if administered routinely for life but can’t actually clear the infection. We are especially excited about our ARCUS-based HBV editing program because it is the first that we are aware of that may offer a functional cure for chronic HBV infection,” said Cassandra Gorsuch, head of gene therapy at Precision BioSciences. “We look forward to submitting an IND/CTA in 2024 so that we can evaluate our approach in the clinic and provide hope to millions of patients in need of a cure.”
Gilead Sciences (Nasdaq:GILD) and Acuitas Therapeutics (Vancouver) collaborated with Precision BioSciences on the research.
“We’re very excited to see this study published in Molecular Therapy and to showcase the compelling reductions in cccDNA and surface antigen obtained with our ARCUS gene-editing platform in two novel animal models of HBV infection,” said Derek Jantz, chief scientific officer of Precision BioSciences, in a news release. “Our data suggest that LNP-delivered ARCUS mRNA is worth further exploration as a possible functional cure for chronic hepatitis B.”
Roughly, 1.5 million people are infected with hepatitis B annually, according to WHO.
Precision BioSciences also highlighted data on the gene-editing technique at the American Society of Gene & Cell Therapy (ASGCT) meeting, held May 16 to 19, in Washington, D.C. The company gave two oral presentations on its PBGENE-HBV and PBGENE-PCSK9 gene editing candidates at the event.
The company’s PBGENE-HBV candidate works to halt virus persistence by targeting covalently closed circular DNA (cccDNA) in chronic hepatitis B patients.
The company anticipates filing paperwork for an investigational new drug (IND) application in 2024.
DTIL shares fell 5.8% today to $1.78.
Precision BioSciences went public in 2019. Its current pipeline comprises ex vivo CAR-T immunotherapy clinical candidates and in vivo gene editing candidates for genetic and infectious diseases.
In late 2021, the company announced a plan to spin out its Elo Life Systems unit, which focuses on the food and agriculture business.
Filed Under: Cell & gene therapy