What's Happening?
Precision BioSciences has announced promising results from its Phase 1 clinical trial of PBGENE-HBV, a gene editing therapy aimed at treating chronic Hepatitis B. The trial, known as ELIMINATE-B, is the first
to target the root cause of the disease by eliminating cccDNA and inactivating integrated HBV DNA. The therapy was well-tolerated across various doses, with no dose-limiting toxicities observed. The trial involved nine patients who received 22 doses across three cohorts, showing dose-dependent antiviral activity. Notably, patients in the highest dose cohort exhibited significant reductions in hepatitis B surface antigen (HBsAg), suggesting a potential path towards a cure.
Why It's Important?
The development of PBGENE-HBV represents a significant advancement in the treatment of chronic Hepatitis B, a condition affecting millions globally. Current treatments only suppress the virus and require lifelong administration, whereas PBGENE-HBV aims to eradicate the virus by targeting its genetic material. This could potentially lead to a functional cure, reducing the burden of the disease and its complications, such as liver cancer and cirrhosis. The success of this trial could pave the way for new gene editing therapies for other viral infections, highlighting the potential of Precision BioSciences' ARCUS platform.
What's Next?
Precision BioSciences plans to complete dosing in the third cohort by early 2026 and explore additional dosing schedules. The company aims to advance PBGENE-HBV into the expansion phase of the ELIMINATE-B study, evaluating the optimized dose regimen in a larger patient population. This phase will include paired biopsies to provide robust evidence of gene editing. The company will host a conference call to discuss these developments and future plans.
