Precision BioSciences, a clinical-stage gene editing company, has announced a significant advancement in its quest to cure chronic hepatitis B. The company has received Clinical Trial Application (CTA) approval in Moldova for its lead candidate, PBGENE-HBV, marking a pivotal step towards a potential cure for the nearly 300 million individuals globally affected by this chronic disease.
This approval paves the way for initiating a Phase 1 clinical program, with patient dosing imminent. Precision BioSciences has adopted a multi-track regulatory strategy, leveraging promising preclinical data to pursue additional CTA and IND approvals internationally. This approach aims to expedite trial enrollment and generate crucial clinical data on both safety and efficacy.
PBGENE-HBV distinguishes itself as the first therapeutic approach to undergo clinical trials designed to eliminate the root cause of chronic hepatitis B – the persistent cccDNA in liver cells. Current standard of care, relying on daily antiviral medication, only offers a 1-3% chance of functional cure. PBGENE-HBV utilizes the company’s proprietary ARCUS® gene editing platform to deliver a specially designed nuclease, via lipid nanoparticles, directly to liver cells. This nuclease targets and eliminates the viral genome, including cccDNA and integrated HBV DNA.
The company plans to present a comprehensive overview of the PBGENE-HBV program on November 15th, preceding the American Association for the Study of Liver Diseases Annual Meeting. This update will provide further insights into the preclinical safety data and Phase 1 trial design.
Chronic hepatitis B is a serious global health concern with limited curative options. A significant proportion of patients develop complications leading to cirrhosis, liver failure, and liver cancer. PBGENE-HBV offers a beacon of hope by directly targeting and eliminating the source of the disease, potentially leading to functional cures and freeing patients from lifelong dependence on antiviral therapies.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
