NEW YORK – Precision BioSciences on Wednesday said it plans to file an investigational new drug application in the US or a clinical trial application in Europe this year as it looks to move its gene-editing candidate for chronic hepatitis B into human studies.
Durham, North Carolina-based Precision BioSciences said it has received feedback, including on a proposed study design and toxicology assessments, from the US Food and Drug Administration and other regulators outside the US on its preclinical strategy to advance the candidate, PBGENE-HBV, into a Phase I trial.
"Based on this feedback, we have initiated the final preclinical studies as well as site selection efforts as we move towards clinical readiness," Precision BioSciences President and CEO Michael Amoroso said in a statement.
PBGENE-HBV is designed to address chronic hepatitis B infection caused by the persistence of hepatitis B virus (HBV) covalently closed circular DNA (cccDNA) and the integration of HBV DNA into patients' liver cells. PBGENE-HBV is based on Precision BioSciences' gene-editing technology, Arcus, which uses sequence-specific DNA-cutting enzymes to insert or eliminate DNA in vivo. Through this, PBGENE-HBV aims to eliminate cccDNA and inactivate the integrated HBV DNA.