Precision BioSciences (DTIL) Jefferies Global Healthcare Conference 2026 summary

Company overview and technology focus

• Specializes in in vivo gene editing using proprietary ARCUS technology, emphasizing unique cut type, size, and simplicity for targeted gene editing applications.

• Current clinical focus is on chronic hepatitis B (HBV) and Duchenne Muscular Dystrophy (DMD) programs.

• Headquartered in Durham, North Carolina, with approximately 100 employees and public since 2019.

HBV program: clinical data and biomarker insights

• Presented groundbreaking data at EASL showing direct elimination of cccDNA in chronic HBV using PBGENE-HBV, with both biopsy and blood marker (pgRNA) evidence.

• pgRNA is highlighted as a specific and predictive biomarker for cccDNA elimination, with growing support for its use over S antigen in guiding safe NUC withdrawal.

• Substantial and durable reductions in S antigen observed, though S antigen is less specific to cccDNA and considered a secondary marker.

• Editing kinetics are rapid, with ARCUS nuclease expression peaking within 1-2 days and complete editing in 1-2 weeks; pgRNA loss seen across all dose levels.

• Over 99% elimination/inactivation of cccDNA demonstrated in biopsies, with durable viral source eradication and reduced risk of hepatocellular carcinoma.

Dose selection and next steps for HBV

• Multiple dose cohorts (0.4, 0.65, 0.8 mg/kg) with various schedules are being evaluated, all showing strong pgRNA eradication.

• Six-month durability of pgRNA elimination is a key criterion for NUC withdrawal; ongoing accrual in key dose cohorts to refine therapeutic window.

• Next steps include stopping NUCs in pgRNA-positive patients with sustained biomarker response, with updates expected later in the year.