Precision BioSciences Unveils New Phase 1 PBGENE-HBV Findings at AASLD The Liver Meeting®, Highlighting Safety, Tolerability, and Dose-Dependent Antiviral Effects in Initial Three Cohorts
PBGENE-HBV Overview: PBGENE-HBV is the first gene editing therapy targeting chronic Hepatitis B by eliminating HBV cccDNA and inactivating integrated HBV DNA, with promising results presented at the AASLD meeting on November 10, 2025.
Clinical Trial Results: The Phase 1 ELIMINATE-B study showed that PBGENE-HBV was well-tolerated across multiple doses, with all patients experiencing significant reductions in hepatitis B surface antigen (HBsAg) and evidence of antiviral activity.
Biopsy Evidence: Paired liver biopsy data confirmed direct gene editing of HBV DNA in patients, marking a significant milestone in demonstrating the therapy's mechanism and potential for a functional cure.
Future Directions: Precision BioSciences plans to complete dosing in the current cohort and expand the study to evaluate optimized dosing regimens, aiming to establish a path toward stopping nucleos(t)ide analogs and testing for a cure.
Get Free Real-Time Notifications for Any Stock
Analyst Views on DTIL
About DTIL
About the author

Precision BioSciences Advances PBGENE-HBV and PBGENE-DMD Trials, Data Updates Expected in 2026
- Clinical Trial Progress: Precision BioSciences' PBGENE-HBV is currently undergoing the Phase 1/2a ELIMINATE-B trial, which has shown dose-dependent antiviral activity, with data updates expected at medical conferences in 2026, potentially offering a cure for chronic hepatitis B patients.
- Strong Financial Position: As of December 31, 2025, Precision anticipates having approximately $137 million in cash and cash equivalents, which is expected to fund key data milestones for PBGENE-HBV and PBGENE-DMD, ensuring operational stability through 2028.
- New DMD Treatment Approach: The PBGENE-DMD Phase 1/2 FUNCTION-DMD trial is expected to receive IND clearance in Q1 2026, aiming to provide durable functional muscle improvement for 60% of DMD patients, with initial data anticipated by the end of 2026.
- Collaborative Project Advancements: The ECUR-506 project with iECURE has reached key alignment with the FDA, supporting its potential as a curative treatment for neonatal OTC deficiency, with clinical trial data expected to be released in the first half of 2026, further enhancing Precision's market position.

iECURE Secures FDA RMAT Designation for ECUR-506, Advancing OTC Deficiency Treatment
- Regulatory Milestone: iECURE's ECUR-506 has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation, which will provide accelerated review and intensive guidance for its development in treating neonatal onset ornithine transcarbamylase deficiency, thereby expediting the clinical development process.
- Clinical Study Alignment: The company has aligned with the FDA on primary and secondary efficacy endpoints, comparators, and sample size for the ongoing OTC-HOPE study, laying the groundwork for a future Biologics License Application (BLA) and showcasing its potential in the rare disease space.
- Innovation Passport: ECUR-506 has also been awarded an Innovation Passport under the UK's Innovative Licensing and Access Pathway (ILAP), making it one of only three selected investigational medicinal products, underscoring its potential to address significant unmet medical needs.
- Clinical Data Support: Encouraging clinical data from the OTC-HOPE study, including a complete clinical response observed in the first treated infant, further validates the efficacy of ECUR-506 and may inform the development of other indications in the pipeline.








