Aug 06, 2026 —
Precision BioSciences reported second-quarter 2026 financial results and provided clinical updates across its in vivo and ex vivo gene editing portfolio, led by new data from PBGENE-HBV, its investigational ARCUS® in vivo gene editing therapy for chronic hepatitis B.
The company highlighted late-breaking data from the ongoing ELIMINATE-B trial, presented at the European Association for the Study of the Liver, or EASL, Congress 2026. Precision described the data as the first clinical biopsy evidence that a therapeutic gene editor can directly target and eliminate cccDNA, the covalently closed circular DNA reservoir that serves as the source of hepatitis B viral replication.
As of the May 4, 2026 data cutoff, 38 doses had been administered across 16 patients in five cohorts. Liver biopsy data showed a 1-log reduction in cccDNA-derived transcripts in one patient after two administrations of PBGENE-HBV at 0.4 mg/kg, with less than 1% of cccDNA remaining after treatment. Analysis from a second patient treated with three doses at the same dose level and schedule suggested that repeat administration may cumulatively increase the anti-cccDNA effect in the liver.
PBGENE-HBV is being evaluated as a potential curative treatment for chronic hepatitis B. The program uses repeat administrations of lipid nanoparticle, or LNP, delivery to enable in vivo gene editing intended to eliminate cccDNA and achieve sustained viral clearance.
Precision also reported sustained loss of pre-genomic RNA, or pgRNA, in 100% of evaluable patients who had detectable pgRNA before treatment. The company described pgRNA as a key blood biomarker for cccDNA elimination because it is produced from cccDNA and contributes to production of new infectious virions.
Additional serum biomarker findings included substantial S-antigen declines in all treated patients, with durability observed across dose levels ranging from 0.2 mg/kg to 0.8 mg/kg. The first patient dosed in ELIMINATE-B continued to show substantial reductions more than one year after dosing.
No dose-limiting toxicities have been observed across 16 patients. Precision said LNP-related hypotension observed during dose escalation was addressed through mitigation measures including longer infusion time and a short course of steroids at infusion.
Beyond hepatitis B, Precision is advancing PBGENE-DMD, its muscle-targeted gene excision program for Duchenne muscular dystrophy. PBGENE-DMD is designed to use two complementary ARCUS nucleases delivered in a single AAV vector to excise exons 45–55 of the dystrophin gene, with the goal of restoring expression of a near full-length dystrophin protein in approximately 60% of patients with DMD.
At the ASGCT 2026 Annual Meeting, the company presented preclinical data showing that PBGENE-DMD treatment in early-juvenile mice produced higher efficacy across key skeletal and respiratory muscles than treatment in late-juvenile mice over a comparable timeframe. Precision said the findings support evaluation in younger DMD patients, including boys ages 2 to 3, who are included in the ongoing FUNCTION-DMD Phase 1/2 trial.
FUNCTION-DMD is now actively recruiting, with clinical sites active at Arkansas Children’s Hospital in Little Rock and Washington University School of Medicine in St. Louis. Initial safety data are expected by year-end 2026.
Precision also provided updates on partnered programs. ECUR-506, an ARCUS-mediated in vivo targeted gene insertion program led by iECURE for neonatal-onset ornithine transcarbamylase deficiency, is being evaluated in the ongoing OTC-HOPE trial. The study is active in the United Kingdom, United States, Australia, and Spain.
In ex vivo cell therapy, azer-cel, an allogeneic CAR-T therapy licensed to Imugene, continues development in diffuse large B-cell lymphoma, with FDA feedback supporting key elements of a potential pivotal study. Separately, azer-cel is being evaluated by TG Therapeutics in a Phase 1 trial for progressive multiple sclerosis.
As of June 30, 2026, Precision reported $112.4 million in cash, cash equivalents, and restricted cash. The company expects its cash balance to support data milestones for both PBGENE-HBV and PBGENE-DMD through 2028, with additional updates from both programs targeted for year-end 2026.
