NEW YORK – Precision BioSciences on Thursday said it has received regulatory permission to begin the first clinical trial of its lead candidate, a gene-editing treatment for chronic hepatitis B, in Moldova.
According to the Durham, North Carolina-based company, it has submitted regulatory applications seeking permission to begin clinical trials of PBGENE-HBV in multiple countries.
With PBGENE-HBV, Precision BioSciences is aiming to cure chronic hepatitis B infection caused by persistent hepatitis B virus (HBV) covalently closed circular DNA (cccDNA) and the integration of HBV DNA into patients' liver cells. Current treatments for hepatitis B are designed to suppress the virus by reducing circulating HBV DNA but these drugs must be taken for life, and they don't eradicate HBV cccDNA, which Precision BioSciences is betting is the "root cause of the disease."
PBGENE-HBV involves delivering a nuclease-encoding mRNA to the liver via lipid nanoparticles using Precision BioSciences' Arcus gene-editing platform. When the mRNA is expressed in HBV-infected hepatocytes, it cuts a sequence in the HBV genome that is designed to eliminate cccDNA in vivo. With this approach, the company hopes to stop HBV from replicating and inactivate integrated HBV DNA in hepatocytes.
The clinical trial in Moldova is just the start of Precision BioSciences' global regulatory strategy for this treatment. The firm said it will continue to submit clinical trial application (CTA) and investigational new drug (IND) filings seeking permission from health authorities to begin Phase I trials for PBGENE-HBV in different countries.
"With rapid regulatory approval in hand, we are working diligently to screen and dose patients at our first clinical site in Moldova," Precision BioSciences CEO Michael Amoroso said in a statement. "In parallel, we are leveraging our robust regulatory package, which highlights the safety and potent antiviral effects of PBGENE-HBV in a variety of models, including nonhuman primates, to pursue additional CTA and IND approvals globally."