Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — Investor Presentation (August 2026)
53 slides from Precision Biosciences's investor presentation, August 2026
Slide 1: Corporate Presentation
Corporate Presentation August 2026
Slide 2: Forward-Looking Statements
Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statement
Slide 3: ARCUS: Precision's Proprietary Gene Editing Platform
ARCUS: Precision’s Proprietary Dedicated to developing novel in Gene Editing Platform vivo gene editing therapeutics with goal of curing difficult-to- 〉 ARCUS wholly owned by Precision BioSciences treat diseases with high unmet 〉 Derived from the homing endonuclease I-CreI found in green algae need,
Slide 4: Multiple Near-Term Data Catalysts Through Wholly Owned Programs
Multiple Near-Term Data Catalysts Through Wholly Owned Programs Clinical-Stage In Vivo Gene-Editing Programs PBGENE-HBV for Chronic Hepatitis B PBGENE-DMD for Duchenne Muscular Dystrophy 〉 Viral gene elimination program with 38 doses delivered across 16 patients 〉 PBGENE-DMD is designed to provide d
Slide 5: Chronic Hepatitis B Multi-Billion Dollar Market Opportunity
Chronic Hepatitis B Multi-Billion Dollar Market Opportunity: Large patient population currently on non-curative treatment options Ch > 300 Million U.S. Market Opportunity of ~$10 Billion1 cHBV infections globally Driven by the drug treated patient population today with total global aggregate revenue
Slide 6: Global DMD Market Poised for Innovative Breakthroughs Like PBGENE-DMD
Global DMD Market Poised for Innovative Breakthroughs Like PBGENE-DMD Prevalence & Incidence1 Global Market Size Projection2 ~300-400k ~15k DMD Patients globally DMD Patients in US ~20% growth $8B rate >20k births ~550 births per year globally per year in US Up to 60% of these patients are eligible
Slide 7: PBGENE-HBV for Chronic Hepatitis B
PBGENE-HBV for Chronic Hepatitis B 7 7
Slide 8: HBV at the Turning Point: From Lifelong Suppression Toward Finite, Biomarker-Guided Viral Cure
HBV at the Turning Point: From Lifelong Suppression Toward Finite, Biomarker-Guided Viral Cure “True cure of HBV “…assessing response to chronic “First clinical evidence infection requires hepatitis B therapies is unclear of reservoir-elimination eradication, degradation because of inconsistent corr
Slide 9: A Direct Antiviral Therapy Targeting & Eliminating cccDNA Is Needed to Cure HBV
A Direct Antiviral Therapy Targeting & Eliminating cccDNA Is Needed to Cure HBV 〉 Current therapies do not eliminate the Current Therapeutics Rarely sole source of infectious particles: cccDNA Achieve Cure Because 〉 Downstream targeting agents only They Do Not Eliminate cccDNA achieve single-digit f
Slide 10: FDA Regulatory Pathway: Elimination of HBV DNA Is Required for Approval
FDA Regulatory Pathway: Elimination of HBV DNA Is Required for Approval PATHWAYS TO FDA APPROVAL1 GUIDANCE DOCUMENT 1 2 Chronic Hepatitis B Virus Infection: OR Developing Drugs for Treatment HBV DNA HBV DNA + APRIL 2022 undetectable HBsAg both only undetectable Loss of HBV DNA Is the Common Therapeu
Slide 11: Phase 1 ELIMINATE-B Trial Efficacy Data
Phase 1 ELIMINATE-B Trial Efficacy Data
Slide 12: Direct Antiviral Targeting: Late-Breaking EASL Data Demonstrate PBGENE-HBV Eliminates cccDNA
Direct Antiviral Targeting: Late-Breaking EASL Data Demonstrate PBGENE-HBV Eliminates cccDNA as Designed, Providing Path Toward Viral Cure Targeting the viral source HBV DNA PBGENE HBV PBGENE-HBV potently eliminates cccDNA as the primary mechanism and pgRNA drove undetectable pgRNA in all patients w
Slide 13: Deep Dive Into PBGENE-HBV Mechanism: Two Productive Editing Outcomes
Deep Dive Into PBGENE-HBV Mechanism: Potently Targeting cccDNA Through Two Productive Editing Outcomes The Reason Why: Predominant Outcome (~90%) Primary PBGENE-HBV targets Mechanism Elimination cccDNA, which is more Replication effective because there are Incompetent two editing outcomes. HBV cccDN
Slide 14: Broadening Patient Experience and Confidence: 38 Doses Across 16 Patients in 5 Cohorts
Broadening Patient Experience and Confidence: 38 Doses Across 16 Patients in 5 Cohorts to Date W0 W4 W8 W12 W16 Patient 1 Cohort 1 0.2 mg/kg Patient 2 Q8W Patient 3 Cohort 2 Patient 4 0.4 mg/kg Patient 5 Q8W Patient 6 Dose Cohort 3 Patient 7 administered 0.8 mg/kg Patient 8 Q8W Patient 9 Dose planne
Slide 15: Multiple Datasets Support cccDNA Targeting and Permanence of Effect by PBGENE-HBV
Multiple Datasets Support cccDNA Targeting and Permanence of Effect by PBGENE-HBV Liver Biopsies pgRNA HBsAg CONFIRMATION OF POTENT cccDNA SUBSTANTIAL ANTIVIRAL cccDNA ELIMINATION EFFECT ACTIVITY IN ALL PATIENTS 〉 10-fold (1-log) reduction in 〉 100% of patients (6/6) with 〉 Durable HBsAg reductions
Slide 16: Multiple Datasets Support cccDNA Targeting and Permanence of Effect by PBGENE-HBV
Multiple Datasets Support cccDNA Targeting and Permanence of Effect by PBGENE-HBV Liver Biopsies pgRNA HBsAg CONFIRMATION OF POTENT cccDNA SUBSTANTIAL ANTIVIRAL cccDNA ELIMINATION EFFECT ACTIVITY IN ALL PATIENTS cccDNA, covalently closed circular DNA; HBsAg, hepatitis B surface antigen; pgRNA, prege
Slide 17: EASL DATA: Biopsies From Two Patients in Cohort 2
EASL DATA: Biopsies From Two Patients in Cohort 2 (0.4 mg/kg, Q8W) Dose 1 Dose 2 Dose 3 Patient 5 Post-treatment Pre-treatment Biopsy Biopsy Patient 6 Post-treatment Biopsy Only Liver Transcript Sequencing Methodology biopsy • Quantitate levels of cccDNA transcripts pre- and post-treatment analyses
Slide 18: EASL DATA: Primary Mechanism - PBGENE-HBV Elimination of cccDNA
EASL DATA: Primary Mechanism - PBGENE-HBV Elimination of cccDNA Resulted in 10-fold (1-log) Decrease in cccDNA 1 Normalized cccDNA transcripts 0.8 10-fold (1-log) Patient 5 decrease 0.6 Patient 5 Cohort 2: 0.4 mg/kg 0.4 Biopsy after 2 administrations Elimination 0.2 <1% Remaining 0 Pre-treatment Pos
Slide 19: EASL DATA: Of the <1% cccDNA Remaining, PBGENE-HBV Indels Inactivate Viral Replication
EASL DATA: Of the <1% cccDNA Remaining, PBGENE-HBV Indels Permanently Inactivate Viral Replication by Knocking Out Polymerase Function 1 Patient 5 | Post dose 2 Normalized cccDNA transcripts 0.8 Elimination 0.6 Key: 0.4 23% Inactivating Indels Wild Type 0.2 <1% Remaining 0 Post-treatment Biopsy Seco
Slide 20: EASL DATA: Additional cccDNA Inactivation Occurs Upon Subsequent Administrations
EASL DATA: Additional cccDNA Inactivation Occurs Upon Subsequent Administrations of PBGENE-HBV Patient 5 | Post dose 2 Patient 6 | Post dose 3 Key: 23% 80% Inactivating Indels Inactivating Edits Wild Type Editing in remaining cccDNA reached 80% after 3 administrations, supporting that cumulative edi
Slide 21: Multiple Datasets Support cccDNA Targeting and Permanence of Effect by PBGENE-HBV
Multiple Datasets Support cccDNA Targeting and Permanence of Effect by PBGENE-HBV Liver Biopsies pgRNA HBsAg CONFIRMATION OF POTENT cccDNA SUBSTANTIAL ANTIVIRAL cccDNA ELIMINATION EFFECT ACTIVITY IN ALL PATIENTS cccDNA, covalently closed circular DNA; HBsAg, hepatitis B surface antigen; pgRNA, prege
Slide 22: Undetectable pgRNA Is Associated With Success of Stopping Nucleoside Analogs
Studies Demonstrate Undetectable pgRNA Is Associated With Success of Stopping Nucleoside Analogs and Increasing Probability of Cure Probability of Cure Stratifies on pgRNA 10X Increase in Probability of Cure With Non-quantifiable pgRNA pgRNA is a specific marker for cccDNA indicating viral load, whe
Slide 23: EASL DATA: PBGENE-HBV Drove Durable pgRNA Loss in 100% of Patients
EASL DATA: PBGENE-HBV Drove Durable pgRNA Loss in 100% of Patients Who Had Detectable pgRNA at Baseline* cccDNA pgRNA HBV DNA Cohort 2, Pt 6 0.4 mg/kg Q8W Cohort 3, Pt 7 0.8 mg/kg Q8W Cohort 3, Pt 9 0.8 mg/kg Q8W Cohort 4, Pt 10 0.4 mg/kg Q4W Cohort 4, Pt 12 0.4 mg/kg Q4W Cohort 5, Pt 14 0.65 mg/kg
Slide 24: Multiple Datasets Support cccDNA Targeting and Permanence of Effect by PBGENE-HBV
Multiple Datasets Support cccDNA Targeting and Permanence of Effect by PBGENE-HBV Liver Biopsies pgRNA HBsAg CONFIRMATION OF POTENT cccDNA SUBSTANTIAL ANTIVIRAL cccDNA ELIMINATION EFFECT ACTIVITY IN ALL PATIENTS Durable HBsAg reductions across all patients treated; highly supportive of cccDNA elimin
Slide 25: EASL DATA: PBGENE-HBV Demonstrates Proof of Editing in 100% of Evaluable Patients
EASL DATA: PBGENE-HBV Demonstrates Proof of Editing in 100% of Evaluable Patients Across All Cohorts, Regardless of Baseline S-Antigen Levels Maximal HBsAg Declines 0 -10 HBsAg % Decline From Baseline -20 -30 Significant maximal -40 declines in HBsAg -50 -60 supporting PBGENE-HBV -70 activity across
Slide 26: EASL DATA: Permanent Editing Effect in First Patient Now >1 Year Since Initiating Treatment
EASL DATA: Permanent Editing Effect in First Patient Treated With PBGENE- HBV Now >1 Year Since Initiating Treatment Patient 1 – Sustained HBsAg Reductions Dose 1 Dose 2 Dose 3 120 600 % of Pre-Treatment HBsAg 100 HBsAg (IU/mL) 80 400 60 40 200 20 0 0 0 7 14 28 42 71 0 7 14 28 Time 42 0 7 14 28 42 5
Slide 27: Phase 1 ELIMINATE-B Trial Safety Data
Phase 1 ELIMINATE-B Trial Safety Data
Slide 28: PBGENE-HBV Safety Profile Executive Summary
PBGENE-HBV Safety Profile Executive Summary 〉 38 doses administered across 16 patients in 5 cohorts 〉 No dose-limiting toxicities observed 〉 Most common adverse events include infusion-related reactions consistent with LNP effects, with onset and resolution within 24 hours of infusion 〉 While transi
Slide 29: Simple and Mechanism-Driven Safety Measures Have Mitigated Hypotension and Liver Lab Abnormalities
Simple and Mechanism-Driven Safety Measures Have Mitigated Hypotension and Liver Lab Abnormalities LNP-inflammatory response 〉 Rapid onset of complement and cytokine elevations after LNP dosing which resolve in 24 hours 〉 LNP-driven transient elevations of complement and cytokines drive hypotension
Slide 30: Summary of PBGENE-HBV Clinical Data
Summary of PBGENE-HBV Clinical Data
Slide 31: Safety and Efficacy Findings Substantiate a Clear Therapeutic Window
Safety and Efficacy Findings Substantiate a Clear Therapeutic Window for Driving Potential Viral Cures in Chronic Hepatitis B 〉 Established primary mechanism of direct cccDNA elimination through liver biopsy, pgRNA, and HBsAg data — 1-log reduction in cccDNA transcripts observed in liver biopsies —
Slide 32: Transforming the Natural History of cHBV Through Direct Viral Targeting of cccDNA
Transforming the Natural History of cHBV Through Direct Viral Targeting of cccDNA CONTINUE STOP NUC EXPAND 〉 Expand number of patients 〉 Work with global investigators to 〉 Select optimal dosing schedule in cohort 4 (0.4 mg/kg) and design next phase of clinical trial for phase 1 expansion and phase
Slide 33: PBGENE-DMD for Duchenne Muscular Dystrophy
PBGENE-DMD for Duchenne Muscular Dystrophy 33 33
Slide 34: DMD is Caused by Mutations in the Dystrophin Gene
DMD is Caused by Mutations in the Dystrophin Gene That Prevent Production of Dystrophin Protein Resulting in Muscle Degeneration Healthy DMD Dystrophin protein is necessary for muscle DMD is caused by mutations in the dystrophin gene maintenance and repair following injury prevent the production of
Slide 35: PBGENE-DMD: Designed to Excise the Hot-Spot Region Between Exons 45-55
PBGENE-DMD: designed to excise the ‘hot-spot’ region between exons 45-55 of the dystrophin gene with goal of permanently and safely restoring muscle function for the majority of patients living with DMD 35 35
Slide 36: Our Approach Has the Potential to Surpass the Current Benchmark for Functional Improvement
Despite the Emergence of Novel Therapies, We Believe our Approach Has the Potential to Surpass the Current Benchmark for Functional Improvement Current Therapies Approved or in PBGENE-DMD: Development Face Multiple Limitations Target Product Profile EXAMPLES OF MICRODYSTROPHIN APPROACHES PBGENE-DMD
Slide 37: PBGENE-DMD Designed to Provide Durable Functional Improvement for 60% of Patients
PBGENE-DMD Designed to Provide Durable Functional Improvement for 60% of Patients with DMD PBGENE-DMD: A single AAV encodes two ARCUS proteins designed to permanently edit a patient’s own DNA sequence, resulting in naturally-expressed, functional dystrophin Up to 60% ARCUS 1 ARCUS 2 of patients have
Slide 38: PBGENE-DMD Designed to Produce a Near Full-Length Dystrophin Protein
PBGENE-DMD Designed to Produce a Near Full-Length Dystrophin Protein, Demonstrated to be Functional in Humans H1 H2 H3 H4 Full-length ABD R1 R2 R3 R4 R5 R6 R7 R8 R9 R10 R11 R12 R13 R14 R15 R16 R17 R18 R19 R20 R21 R22 R23 R24 CR CT Dystrophin Full-length Dystrophin Functional ABD H1 R1 R2 R3 H2 R4 R5
Slide 39: Near Full-Length Dystrophin Protein Has Known Function in Del45-55 Genotype
Near Full-Length Dystrophin Protein Has Known Function in Individuals with Dystrophin Del45-55 Genotype Lifespan: Lifespan: • Early death in late teens or 20s • Can live into 60-70s1-3 Clinical Presentation: Clinical Presentation: • Progressive muscle weakness leading • Asymptomatic or mild to loss
Slide 40: ~5% Dystrophin is Associated with Meaningful Clinical Benefit
~5% Dystrophin is Associated with Meaningful Clinical Benefit No dystrophin (0%) Group A (n=42) Residual dystrophin (0-5%) Group B (n=34) Residual dystrophin (≥5%) Group C (n=14) 〉 ~5% dystrophin associated with prolonged ambulation beyond typical loss of ambulation time (~10–12 years)and linked to
Slide 41: PBGENE-DMD Resulted in Increased Dystrophin Protein Levels Over Time
PBGENE-DMD Resulted in Increased Dystrophin Protein Levels Over Time, Exceeding Expected Therapeutic Threshold Heart Calf Quadriceps 15 30 25 Dystrophin Restoration (%) Dystrophin Restoration (%) Dystrophin Restoration (%) 25 20 10 20 15 15 10 5 10 5 5 0 0 0 Treated Treateddisease mice disease mice
Slide 42: NEW ASGCT DATA: Therapeutic Dystrophin Restoration in Respiratory Muscles of Early Juvenile Mice
NEW ASGCT DATA: PBGENE-DMD Achieves Therapeutic Dystrophin Restoration in Critical Respiratory Muscles of Early Juvenile DMD Mice Diaphragm Intercostals 10 25 30 Dystrophin protein restoration in Dystrophin Positive Fibers (%) early juvenile mice was up to Dystrophin Restoration (%) Dystrophin Resto
Slide 43: NEW ASGCT DATA: Broad Distribution of Dystrophin Positive Cells
NEW ASGCT DATA: Broad Distribution of Dystrophin Positive Cells Across Skeletal, Cardiac, and Respiratory Muscles After PBGENE-DMD Treatment PBGENE-DMD Heart Calf Quadriceps Disease Model 75 80 60 Dystrophin positive cells (%) Dystrophin positive cells (%) Dystrophin positive cells (%) 60 60 45 45 4
Slide 44: PBGENE-DMD Significantly Improved Muscle Function and Demonstrated Potential for Long-Term Durability
PBGENE-DMD Significantly Improved Muscle Function and Demonstrated Potential for Long-Term Durability Untreated Untreated PBGENE-DMD PBGENE-DMD Diseased Healthy (1x1014 vg/kg) (3x1013 vg/kg) 200% Normalized Force/BW 150% to Untreated Disease 100% 50% 0% 〉Improved muscle function observed from 3 to 6
Slide 45: FUNCTION-DMD Phase 1/2 Clinical Trial
FUNCTION-DMD Phase 1/2 Clinical Trial 45
Slide 46: Phase I/II Study Designed for Safety and Speed
Phase I/II Study Designed for Safety and Speed Clinical Trial Identifier: NCT07429240 Inclusion criteria • Male age 2-7 • Stable IMR regimen ≥12 weeks • Able to complete age-appropriate • DMD with mutations in exons 45-55 motor testing assessments • Up-to-date vaccines Part 1: Part 2: Initial Safety
Slide 47: Phase I/II Study Endpoints
Phase I/II Study Endpoints Clinical Trial Identifier: NCT07429240 Primary Endpoints 〉 Incidence, severity, and causality of TRAEs and SAEs from dosing through Week 104 〉 AEs and SAEs that occur or worsen after initiation of the investigational treatment Secondary Endpoints Exploratory Endpoints Biol
Slide 48: Key Inclusion Criteria
Key Inclusion Criteria Category Inclusion Criteria Age / Sex Males, 2–7 years of age (inclusive) at time of consent Diagnosis Genetically confirmed DMD with mutations in exons 45–55 Able to complete age-appropriate motor testing assessments • Ages 2–<4: walk ≥10 meters Motor Ability • Ages 4–7: Must
Slide 49: Safety as Top Priority: Immune Modulation Regimen (IMR) Strategy
Safety as Top Priority: Immune Modulation Regimen (IMR) strategy ensures monitoring and interventions are optimized to patient safety needs during two key safety risk periods Safety Mitigation: Preventing early and late serious immune complications. AAV therapies can trigger two predictable immune-r
Slide 50: World-Class Clinical Network with Proven Duchenne Expertise
World-Class Clinical Network with Proven Duchenne Expertise 〉 Deep AAV gene therapy expertise across Phase 1–3, including immune risk management and pediatric neuromuscular dosing 〉 Elite Duchenne trial network anchored by PPMD-Certified Care Centers and MDA Care Clinics with demonstrated, reliable
Slide 51: Summary and Financials
Summary and Financials my
Slide 52: Multiple Near-Term Data Catalysts Supported Through Cash Runway
Multiple Near-Term Data Catalysts Supported Through Cash Runway PBGENE-HBV for Chronic Hepatitis B PBGENE-DMD for Duchenne Muscular Dystrophy 〉 Viral gene elimination program with 38 doses delivered across 16 patients 〉 PBGENE-DMD is designed to provide durable functional muscle treated in 5 cohorts
Slide 53: Precision BioSciences Leadership Team Driving Programs Through Next Clinical Milestones
Precision BioSciences Leadership Team Driving Programs Through Next Clinical Milestones Michael Amoroso Cassie Gorsuch, Ph.D. Alex Kelly Naresh Tanna Dario Scimeca Since Oct 2021 Since June 2016 Since Oct 2020 Since Jan 2022 Since June 2019 President & Chief Scientific Officer Chief Operating Office
Precision BioSciences — Investor Presentation (August 2026) on 6ix