Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — AASLD 2025
19 slides from Precision Biosciences's conference presentation, Nov 10, 2025
Slide 1: PBGENE-HBV, a First-in-class Gene Editing Therapy for Chronic Hepatitis B
AASLD 2025 Publication Number: 5017 PBGENE-HBV, a First-in-class Gene Editing Therapy for Chronic Hepatitis B, Demonstrates Safety and Antiviral Activity in Early Cohorts Man-Fung Yuen1, Alina Jucov2,3, Edward Gane4, Emily B. Harrison5, Andrew Van Cott5, Abhishek Chandiramani5, Neil Leatherbury5, Jo
Slide 2: Man-Fung Yuen Disclosures
Man-Fung Yuen Disclosures 〉Advisory committee member: Aligos Therapeutics, AiCuris, Arbutus Biopharma, Clear B Therapeutics, Fujirebio Incorporation, GlaxoSmithKline, Gilead Sciences, Immunocore, Janssen, Roche, Sysmex Corporation, Tune Therapeutics, Vir Biotechnology and Visirna Therapeutics 〉Speak
Slide 3: HBV Cure Requires a Novel Approach Targeting the Viral Replication Source
HBV Cure Requires a Novel Approach Targeting the Viral Replication Source Eliminating cccDNA has a Clear Biologic Rationale for Cure PBGENE "The ideal therapeutic strategy for HBV 1 Eliminates cccDNA curative approaches includes reduction or elimination of the whole cccDNA pool." —Ligat et al. 2020
Slide 4: PBGENE-HBV: First in Class Gene Editor Designed To Eliminate cccDNA
PBGENE-HBV: First in Class Gene Editor Designed To Eliminate cccDNA and Inactivate Integrated HBV DNA PBGENE-HBV is a lipid nanoparticle (LNP) delivered mRNA encoding an ARCUS gene editing nuclease that uniquely eliminates the root cause of HBV with curative intent 〉 PBGENE-HBV specifically recogniz
Slide 5: PBGENE-HBV Preclinical Data Demonstrates cccDNA Elimination and Integrated HBV DNA Inactivation
PBGENE-HBV Preclinical Data Demonstrates cccDNA Elimination and integrated HBV DNA Inactivation cccDNA Elimination Integrated HBV DNA Inactivation (NHP AAV Model) (HBV Transgenic Mouse Model) Cumulative editing with 99% viral editing after two doses Significant HBV DNA reduction with no viral reboun
Slide 6: Phase 1 Study Objectives
Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in HBeAg Negative Patients With Chronic Hepatitis B (NCT06680232) Objectives Primary Objective: Secondary Objectives: 〉To evaluate the safety of PBGENE-HBV in 〉To further evaluate the safety and tolerability participants with Chro
Slide 7: Phase 1 Study Endpoints
Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in HBeAg Negative Patients With Chronic Hepatitis B (NCT06680232) Endpoints Primary Endpoint Secondary Endpoints Safety determined by: Additional safety determined by: 〉 Frequency and severity of dose-limiting toxicities (DLTs) 〉
Slide 8: Key Inclusion / Exclusion Criteria
Key Inclusion / Exclusion Criteria Inclusion Criteria Exclusion Criteria • HBeAg-negative cHBV (~80% patients on NUCs1) • No history of liver cirrhosis regardless of any subsequent improvement in histology • cHBV infection documented by serum HBsAg-positivity for ≥ 12 months • No hepatitis A virus i
Slide 9: Phase 1 Study Design
Phase 1 Study Design (NCT06680232) Part 1: Multiple Ascending Dose Escalation Part 2: Dose Expansion Finite Treatment: Patient receives up to 3 dose administrations Advance optimized dose and schedule to eliminate cccDNA and drive cure Additional Cohorts if needed Cohort 3 | 0.8 mg/kg Go Forward Dos
Slide 10: Demographics and Baseline Characteristics
Demographics and Baseline Characteristics Sex Number Percent Male 10 100 Ethnicity/Race Caucasian 5 50 Asian 4 40 Native Hawaiian 1 10 or Other Pacific Islander Mean Range Age (years) 51 39 - 66 Time with HBV (years) 22 7 - 39 Time on NUCs (years) 10 4 - 25 Baseline HBsAg (IU/mL) 2,217* 370 - 11,813
Slide 11: PBGENE-HBV Safety: Treatment Related Adverse Events
Data cut: Oct 31st, 2025 PBGENE-HBV Safety: Treatment Related Adverse Events Patients Experiencing Treatment Related Adverse Events Events Occurring in at Least 2 Patients or ≥ Grade 3 (n =10) Preferred Term All Grades (%) Grade 3 (%) Pyrexia 9 (90) 0 (0) No DLTs have been observed Chills 8 (80) 0 (
Slide 12: PBGENE-HBV Hepatic Safety Lab Results Across Cohorts
Data cut: Oct 31st, 2025 SAFETY: PBGENE-HBV Hepatic Safety Lab Results Across cohorts ALT elevations <5X ULN with no Transient AST elevations Bilirubin fluctuations sustained elevations resolved quickly near normal range 120 500 500 10X ULN 10X ULN 450 450 100 400 400 Bilirubin fluctuations in Patie
Slide 13: HBsAg Results for Cohort 1 (0.2 mg/kg)
Data cut: Oct 31st, 2025 EFFICACY: PBGENE-HBV HBsAg Results for Cohort 1 (0.2 mg/kg) Patient 1 Patient 2 Patient 3 (Deepest HBsAg reduction: 56%, 0.36log) (Deepest HBsAg reduction: 69%, 0.51log) (Deepest HBsAg reduction: 47%, 0.28log) 800 14000 1200 700 12000 1000 600 10000 800 500 HBsAg (IU/mL) HBs
Slide 14: HBsAg Results for Cohort 2 (0.4 mg/kg)
Data cut: Oct 31st, 2025 EFFICACY: PBGENE-HBV HBsAg Results for Cohort 2 (0.4 mg/kg) Patient 4 Patient 5 Patient 6 (Deepest HBsAg reduction: 59%, 0.39log) (Deepest HBsAg reduction: 66%, 0.47log) (Deepest HBsAg reduction: 44%, 0.25log) 1600 3500 1200 1400 3000 1000 1200 2500 800 1000 HBsAg (IU/mL) HB
Slide 15: HBsAg Results for Cohort 3 (0.8 mg/kg)
Data cut: Oct 31st, 2025 EFFICACY: PBGENE-HBV HBsAg Results for Cohort 3 (0.8 mg/kg) Patient 7 Patient 8 Patient 9 (Deepest HBsAg reduction: 62%, 0.41log) (Deepest HBsAg reduction: 55%, 0.35log) (Deepest HBsAg reduction: 19%, 0.09log) 700 450 500 400 450 600 400 350 500 350 300 300 299 IU/mL HBsAg (
Slide 16: HBsAg Lab Results Across Cohorts
Data cut: Oct 31st, 2025 Efficacy: PBGENE-HBV HBsAg Lab Results Across Cohorts Cohort 1 | 0.2 mg/kg Cohort 2 | 0.4 mg/kg Cohort 3 | 0.8 mg/kg Activity in 3 of 3 patients Activity in 3 of 3 patients Activity in 3 of 3 patients Durable response in 1 of 3 patients Durable response in 3 of 3 patients Do
Slide 17: Proof of Mechanism: Evidence of PBGENE-HBV editing viral DNA
Proof of Mechanism: Evidence of PBGENE-HBV editing viral DNA Preclinical NHP Viral DNA Editing Clinical Biopsy Data from Patient #5 (Two Admins at 0.5 mg/kg) (Two Admins at 0.4 mg/kg) % Viral DNA Editing 100 Inactivation with indels 80 28% in remaining viral DNA 60 40 Elimination measured by loss Da
Slide 18: Conclusions
AASLD 2025 Publication Number: 5017 Conclusions Safety: Repeat dosing designed to drive cumulative 〉 PBGENE-HBV at doses as high as 0.8 mpk administered antiviral effects and cure every 8 weeks has not resulted in a DLT in the initial 3 dose cohorts tested 700 Dose #1 〉 Repeat administrations were w
Slide 19: Acknowledgements
We thank the study participants, investigators, and clinical site staff for their participation and support in the ELIMINATE-B study. We also acknowledge the important contributions of the clinical operations and other functional teams, as well as our partners in the HBV field who have shared valuab