PBGENE-HBV / Precision BioSci - LARVOL DELTA

Initial safety data from ELIMINATE-B, the first clinical trial of a gene editing treatment for chronic hepatitis B (EASL 2025) - "Clinical safety data demonstrates that PBGENE-HBV was well tolerated with no DLTs and no serious adverse events. In combination with PK data, this validates the translation of the NHP model to the clinic, supporting the evaluation of multiple dose administrations and dose levels of PBGENE-HBV to achieve cumula- tive editing in support of cure. Comprehensive datasets of the tolerability and efficacy of multiple administrations are being collected and will be analyzed on full dosing of cohorts."

Clinical • Late-breaking abstract • Fibrosis • Hepatitis B • Hepatocellular Cancer • Hepatology • Infectious Disease • Inflammation • Oncology • Solid Tumor

Initial safety data from ELIMINATE-B, the first clinical trial of a gene editing treatment for chronic hepatitis B (ASGCT 2025) - "In culmination, the initial clinical safety data discussed above demonstrates the translatability of the NHP model and supports the continued evaluation of multiple administrations and dose levels of PBGENE-HBV with the goal of cure. Disease Focus of Abstract:Hepatitis"

Clinical • Cardiovascular • Fibrosis • Hematological Disorders • Hepatitis B • Hepatocellular Cancer • Hepatology • Infectious Disease • Inflammation • Oncology • Portal Hypertension • Solid Tumor

Precision BioSciences Receives U.S. FDA Fast Track Designation for PBGENE-HBV, a First-In-Class Gene Editing Therapy Designed to Eliminate the Root Cause of Chronic Hepatitis B (Businesswire) - "Precision BioSciences, Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for PBGENE-HBV, the Company’s lead wholly owned in vivo gene editing program designed to cure chronic hepatitis B by eliminating cccDNA, the key source of replicating hepatitis B virus (HBV), and inactivating integrated HBV DNA in hepatocytes....Precision is evaluating PBGENE-HBV in the ongoing global Phase 1 ELIMINATE-B trial, with clinical investigation in the United States, Moldova, Hong Kong, New Zealand, and the United Kingdom. The company anticipates sharing updates on the full low-dose cohort, including multiple dose administrations, and data from higher dose levels throughout 2025."

Fast track • P1 data • Hepatitis B

Precision BioSciences Presents Preclinical Safety and Efficacy Data Supporting Repeat Dosing of PBGENE-HBV With a Goal of Curing Chronic Hepatitis B at the Global Hepatitis Summit 2025 (Businesswire) - "Precision BioSciences, Inc...presents preclinical data supporting repeat dosing of PBGENE-HBV in the ELIMINATE-B trial to treat patients with chronic HBV. Even with today’s standard of care, an estimated 15% to 40% of patients with HBV infections may develop complications...These data will be presented during an oral presentation at the Global Hepatitis Summit (GHS) 2025, being held March 18-21, 2025, in Los Angeles, California...In non-human primates (NHPs), repeated administration of PBGENE-HBV was safe and well tolerated, with no systemic accumulation of PBGENE-HBV drug product. Furthermore, PBGENE-HBV was not distributed to germ cells at any of the analyzed timepoints, supporting the conclusion that there is no risk of heritable genome edits."

Preclinical • Hepatitis B

Precision BioSciences Announces Clearance of Investigational New Drug Application by the U.S. FDA for First-in-Class PBGENE-HBV Designed to Eliminate Root Cause of Chronic Hepatitis B (Businesswire) - "Precision BioSciences, Inc...announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for PBGENE-HBV.... Company to expand its Phase 1 ELIMINATE-B study to the U.S., joining world-class clinical sites in Moldova, Hong Kong, and New Zealand where strong clinical execution is currently underway....Precision plans to share detailed clinical data throughout 2025."

IND • P1 data • Trial status • Hepatitis B

Precision BioSciences Announces Initial Safety and Antiviral Activity of PBGENE-HBV in the ELIMINATE-B Clinical Trial (Businesswire) - P1 | N=45 | ELIMINATE-B (NCT06680232) | Sponsor: Precision BioSciences, Inc. | "ELIMINATE-B Phase 1 dose finding study for chronic Hepatitis B executing on schedule with completion of first dose administration for cohort 1 (n=3 patients); PBGENE-HBV, the first LNP gene editing technology studied for Hepatitis B, was safe and well tolerated; PBGENE-HBV demonstrated substantial antiviral activity measured by reduction of Hepatitis B surface antigen (HBsAg) after one administration at the lowest dose level; First clinical proof-of-concept in chronic Hepatitis B for a unique editing modality designed to directly eliminate and inactivate the root cause of Hepatitis B virus from covalently closed circular DNA (cccDNA) and integrated DNA; These PBGENE-HBV data mark the second clinical validation for ARCUS in vivo gene editing in 2025..."

P1 data • Hepatitis B

ELIMINATE-B: Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients with Chronic Hepatitis B (clinicaltrials.gov) - P1 | N=45 | Recruiting | Sponsor: Precision BioSciences, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Hepatitis B • Hepatology • Infectious Disease • Inflammation

ELIMINATE-B: Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients with Chronic Hepatitis B (clinicaltrials.gov) - P1 | N=45 | Not yet recruiting | Sponsor: Precision BioSciences, Inc.

New P1 trial • Hepatitis B • Hepatology • Infectious Disease • Inflammation

Preclinical safety data for PBGENE-HBV gene editing program supports advancement to clinical trials as a potentially curative treatment for chronic hepatitis B (EASL-ILC 2024) - "ARCUS is a single-component gene editor that has been engineered to specifically cut HBV DNA leading to elimination of cccDNA and inactivation of integrated HBV DNA without impacting any sites in the human genome. LNP delivery of ARCUS mRNA was well tolerated in nonhuman primates over multiple administrations. Robust preclinical safety data supports the advancement of PBGENE-HBV to clinical trials as a potential curative, finite treatment for HBV."

Late-breaking abstract • Preclinical • Genetic Disorders • Hepatitis B • Hepatology • Infectious Disease • Inflammation

Precision BioSciences to Present Preclinical Gene Editing Data on its ARCUS-Based Chronic Hepatitis B Program at the HEP DART 2021 Conference (Businesswire) - "Precision BioSciences, Inc...announced it will present preclinical data for its chronic hepatitis B virus (HBV) program at the HEP DART 2021 conference taking place in Cabo San Lucas, Mexico, December 5 – 9, 2021...'We look forward to building on this research with our PBGENE-HBV gene editing candidate to pursue a potential one-time, permanent treatment for chronic hepatitis B and expect to submit an IND application or CTA in 2024.'"

IND • Preclinical • Hepatitis B • Infectious Disease

Precision BioSciences Outlines Clinical Development Strategy for In Vivo Gene Editing Pipeline (Businesswire) - "Precision will pursue clinical development of its PBGENE-HBV candidate for chronic hepatitis B virus (HBV) and expects to submit an IND/CTA in 2024....New preclinical data to be presented today, and data previously presented at the American Society of Gene & Cell Therapy Annual Meeting, show that ARCUS efficiently targeted and degraded HBV cccDNA in HBV-infected primary human hepatocytes and reduced expression of HBV S-antigen (HBsAg) by as much as 95%. Similar levels of HBsAg reduction were observed in a newly developed mouse model of HBV infection following administration of ARCUS mRNA using lipid nanoparticle (LNP) delivery."

IND • Preclinical • Hepatitis B • Infectious Disease