Precision BioSciences, Inc. · NASDAQ:DTIL
Precision BioSciences — AASLD 2026
74 slides from Precision BioSciences's conference presentation, Nov 5, 2026
Slide 1: AASLD 2026 Investor Update: Direct cccDNA Elimination Leads to Potential Viral Cure
AASLD 2026 Investor Update: Direct cccDNA Elimination Leads to an Era of Potential Chronic Hepatitis B Viral Cure Confidential
Slide 2: Forward-Looking Statements
Forward-Looking Statements 2 This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this presentation that do not relate to matters of historical fact should be considered forward-looking statement
Slide 3: Setting the Stage for Viral Cure
Setting the Stage for Viral Cure
Slide 4: The Problem: cccDNA is the root cause of chronic hepatitis B
4 ASO, antisense oligonucleotide; CAM, capsid assembly modulator; cccDNA, covalently closed circular DNA; HBV, hepatitis B virus; mAb, monoclonal antibody; MOA, mechanism of action; NUC, nucleos(t)ide analog; siRNA, small interfering RNA. The Problem: cccDNA is the root cause of chronic hepatitis B
Slide 5: Since May 2026 at EASL
Since May 2026 at EASL… cccDNA, covalently closed circular DNA; EASL, European Association for the Study of the Liver; pgRNA, pregenomic RNA. 5 Treatment Effect in 100% of Patients 38 administrations, 16 patients From the Liver to the Blood Advent of pgRNA blood biomarker 100% of patients with durab
Slide 6: AASLD Late-Breaking Oral Presentation Demonstrating cccDNA Elimination at 0.4 mg/kg
-4 0 4 8 12 16 20 24 28 32 36 40 44 48 52 56 60 Weeks from First Dose Pt 6 0.4 mg/kg Pt 7 0.8 mg/kg Pt 10 0.4 mg/kg Pt 14 0.65 mg/kg Pt 9 0.8 mg/kg Pt 12 0.4 mg/kg Pt 15 0.65 mg/kg Pt 16 0.65 mg/kg Pt 18 0.4 mg/kg Pt 21 0.4 mg/kg Pt 19 0.4 mg/kg Pre doseAASLD Late-Breaking Oral Presentation at AASLD
Slide 7: 100% pgRNA Loss in the First HBeAg+ Patient
AASLD, American Association for the Study of Liver Diseases; cccDNA, covalently closed circular DNA; HBeAg, hepatitis B e antigen; HBV, hepatitis B virus; pgRNA, pregenomic RNA. Data cutoff: September 28, 2026. 7 Dose 1 Dose 2 Limit of Detection0 1000 2000 3000 4000 5000 6000 -14 0 14 28 42 56 70 HB
Slide 8: Establishing PBGENE-HBV as the Foundation of Treatment in HBV
Establishing PBGENE-HBV as the Foundation of Treatment in HBV: ELIMINATE-B Trial Moves to Phase 2 Expansion PBGENE-HBV 0.4 mg/kg Q8W Viral Cure cccDNA Elimination Endpoint: Undetectable HBV DNA off therapy Functional Cure Suppression of viral infection Endpoint: Undetectable HBV DNA and HBsAg off th
Slide 9: PBGENE-HBV: Eliminating cccDNA to Drive Viral Cure
PBGENE-HBV: Eliminating cccDNA to Drive Viral Cure
Slide 10: cccDNA Is the Only Source of New Infectious Particles
cccDNA Is the Only Source of New Infectious Particles Known as HBV DNA 10 cccDNA in liver pgRNA in blood HBV DNA in blood (Infectious particles) cccDNA, covalently closed circular DNA; HBV, hepatitis B virus; pgRNA, pregenomic RNA. The Trinity of HBV Infection cccDNA makes pgRNA makes HBV DNA Theref
Slide 11: While on NUCs, Patients Have Undetectable HBV DNA
While on NUCs, Patients in ELIMINATE-B Have Undetectable HBV DNA 11 cccDNA in liver pgRNA in blood HBV DNA in blood (Infectious particles) cccDNA is only present in the liver and requires biopsies Therefore, pgRNA is the biomarker in the blood for HBV DNA NUC cccDNA, covalently closed circular DNA;
Slide 12: From Decades to Days: PBGENE-HBV Eliminates cccDNA
From Decades to Days: PBGENE-HBV Eliminates cccDNA, Potentially Changing the Natural History of Chronic Hepatitis B Enabling a Viral Cure 12 cccDNA in liver pgRNA in blood HBV DNA in blood (Infectious particles) By directly eliminating cccDNA, PBGENE-HBV is designed to eradicate the trinity of viral
Slide 13: Phase 1 ELIMINATE-B Data Support Advancement to Phase 2
Phase 1 ELIMINATE-B Data Support Advancement to Phase 2
Slide 14: Patient Baseline Characteristics (n=25)
Patient Baseline Characteristics (n=25) 14 C/mL, copies per milliliter; HBeAg, hepatitis B e antigen; HBsAg, hepatitis B surface antigen; IU/mL, international units per milliliter; NUC, nucleos(t)ide analog; pgRNA, pregenomic RNA; TND, target not detected. Screening HBsAg value used for Patient 25,
Slide 15: 59 Administrations in 25 Patients Across 4 Dose Levels
15 HBV, hepatitis B virus; Pt; patient. Data cutoff: September 28, 2026. Comprehensive Evaluation of PBGENE-HBV: 59 Administrations in 25 Patients Across 4 Dose Levels 0.4 mg/kg0.2 mg/kg 0.8 mg/kg0.65 mg/kg
Slide 16: Extensive Experience with the Go-Forward Dose 0.4 mg/kg
16 HBV, hepatitis B virus; Pt; patient. Data cutoff: September 28, 2026. Extensive Experience with the Go-Forward Dose 0.4 mg/kg PBGENE-HBV 3 pts 9 admins 3 pts 7 admins 6 pts 13 admins 13 pts 30 admins 0.4 mg/kg0.2 mg/kg 0.8 mg/kg0.65 mg/kg Achieved Target Product Profile for PBGENE-HBV as Low as 0
Slide 17: PBGENE-HBV Drives Durable pgRNA Undetectable Through cccDNA Elimination
-4 0 4 8 12 16 20 24 28 32 36 40 44 48 52 56 60 Weeks from First Dose NEW AASLD DATA: PBGENE-HBV Drives Ongoing Durable pgRNA Undetectable Through cccDNA Elimination 17 Roche cobas® HBV RNA test limit of detection 3.3-5 copies/mL, limit of quantitation 10 copies/mL, https://pubmed.ncbi.nlm.nih.gov/3
Slide 18: pgRNA Loss in 100% of Patients at 0.4 mg/kg
Patient 6 0.4 mg/kg Patient 10 0.4 mg/kg Patient 12 0.4 mg/kg Patient 18 0.4 mg/kg Patient 21 0.4 mg/kg Patient 19 0.4 mg/kg -4 0 4 8 12 16 20 24 28 32 36 40 44 48 52 56 60 Weeks from First Dose NEW AASLD DATA: PBGENE-HBV Drives Ongoing Durable pgRNA Undetectable Through cccDNA Elimination 18 cccDNA
Slide 19: HBeAg Positive Patients: Most Difficult to Treat
HBeAg Positive Patients: The Most Difficult to Treat with Greatest cccDNA Burden The median cccDNA levels were 2-3log higher in HBeAg+ patients compared to HBeAg- patients. — Werle-Lapostolle et al, 2004, Gastroenterology
Slide 20: Viral Cure Is Hardest to Achieve in HBeAg Positive Patients
A Viral Cure is Hardest to Achieve in HBeAg Positive Patients Because They Have Logarithmically More cccDNA and Therefore More pgRNA 20 cccDNA in liver pgRNA in blood HBeAg NegativeHBeAg Positive pgRNA in bloodcccDNA in liver cccDNA, covalently closed circular DNA; HBeAg, hepatitis B e antigen; pgRN
Slide 21: Eliminating cccDNA at 0.4 mg/kg Leads to 100% Loss of pgRNA
Dose 1 Dose 2 0 1000 2000 3000 4000 5000 6000 -14 0 14 28 42 56 70 HB V pg RN A (C /m L) Days from First Dose HBV pgRNA: Patient 19 NEW AASLD DATA: Eliminating cccDNA at 0.4 mg/kg Leads to 100% Loss of pgRNA in First HBeAg+ Patient 100% Loss of pgRNA in First HBeAg Positive Patient 0 pgRNA loss in f
Slide 22: Liver Biopsy Evidence Supports cccDNA Elimination at 0.4 mg/kg
Liver Biopsy Evidence Supports cccDNA Elimination at 0.4 mg/kg
Slide 23: Biopsy Data Reproduced Potent cccDNA Elimination at 0.4 mg/kg
NEW AASLD DATA: Biopsy Data Reproduced Potent cccDNA Elimination at 0.4 mg/kg 23 AASLD, American Association for the Study of Liver Diseases; cccDNA, covalently closed circular DNA. Data cutoff: September 28, 2026. 0 0.2 0.4 0.6 0.8 1 First Biopsy Second Biopsy No rm al iz ed c cc DN A tr an sc ri p
Slide 24: Secondary Biomarker HBsAg: Every Patient Experienced Treatment Effect
Secondary Biomarker HBsAg: Every ELIMINATE-B Patient Experienced Treatment Effect with PBGENE-HBV (n=24 evaluable at time of data cutoff)
Slide 25: Substantial HBsAg Declines in All Patients
25 The 3 patients with only 1 dose administered to date are detectable for pgRNA at baseline, but not yet evaluable for pgRNA. Patient 25 is not yet evaluable for efficacy. AASLD, American Association for the Study of Liver Diseases; cccDNA, covalently closed circular DNA; HBsAg, hepatitis B surface
Slide 26: 0.4 mg/kg is Sufficient for Maximal Efficacy and Durability
26 Patient 25 is not yet evaluable for efficacy. AASLD, American Association for the Study of Liver Diseases; HBsAg, hepatitis B surface antigen. Data cutoff: September 28, 2026. Maximal HBsAg declines observed as low as 0.4 mg/kg -100% -90% -80% -70% -60% -50% -40% -30% -20% -10% 0% M ax D ec lin e
Slide 27: Definitive Phase 1 Efficacy Data Support 0.4 mg/kg Go-Forward Dose
0 0.2 0.4 0.6 0.8 1 First Biopsy Second Biopsy No rm al iz ed c cc DN A tr an sc ri pt s Patient 6 at AASLD 35-fold (1.5-log) decrease NEW AASLD DATA: Definitive Phase 1 Efficacy Data Support 0.4 mg/kg as Go-Forward Dose AASLD, American Association for the Study of Liver Diseases; cccDNA, covalently
Slide 28: PBGENE-HBV Safety Profile Executive Summary
PBGENE-HBV Safety Profile Executive Summary 28 ALT, alanine aminotransferase; AST, aspartate aminotransferase; HBV, hepatitis B virus; LNP, lipid nanoparticle. Data cutoff: September 28, 2026. 1. All data from 0.8 mg/kg dose level presented in May 2026 Investor Presentation. No additional doses or s
Slide 29: Safety Profile: Clear Evidence of Therapeutic Window at 0.4 mg/kg
PBGENE-HBV Safety Profile: Clear Evidence of Therapeutic Window at 0.4 mg/kg AE, adverse event; ALT, alanine aminotransferase; AST, aspartate aminotransferase; DMC, data monitoring committee; HBV, hepatitis B virus; LNP, lipid nanoparticle; SAE, serious adverse event. Data cutoff: September 28, 2026
Slide 30: Go-Forward Phase 2 Dose of 0.4 mg/kg Selected Based on Evidence
Go-Forward Phase 2 Dose of 0.4 mg/kg for PBGENE-HBV: Selected Based on Compelling Evidence Efficacy: 100% of patients experienced a treatment effect with PBGENE-HBV (n=24) 92% (11/12) of patients achieved pgRNA loss to undetectable Last remaining patient had 95% pgRNA reduction at data cutoff with a
Slide 31: What's Next: PBGENE-HBV Moves to Phase 2 Global Expansion
What’s Next: PBGENE-HBV Moves to Phase 2 Global Expansion
Slide 32: ELIMINATE-B Trial Moves to Phase 2 Expansion
Establishing PBGENE-HBV as the Foundation of Treatment in HBV: ELIMINATE-B Trial Moves to Phase 2 Expansion PBGENE-HBV 0.4 mg/kg Q8W Viral Cure cccDNA Elimination Endpoint: Undetectable HBV DNA off therapy Functional Cure Suppression of viral infection Endpoint: Undetectable HBV DNA and HBsAg off th
Slide 33: PBGENE-HBV Goal to Improve Upon Cure Rates in CHB
Viral Cure rate today: 0% Goal for PBGENE-HBV: Enable first ever viral cure Functional Cure rate today: 19% (bepirovirsen*)2 Goal in the field: 30% functional cure3 PBGENE-HBV Goal to Improve Upon Cure Rates in CHB Viral Cure Complete cccDNA elimination in the liver FDA Endpoint: Undetectable HBV DN
Slide 34: Rationale for Potential Combination
Rationale for Potential Combination
Slide 35: Bepirovirsen Most Effective in Patients with Lower Viral Burden
Bepirovirsen Most Effective in Patients with Lower Viral Burden; 19% Functional Cure Rate in B-Well 1 and B-Well 2 Studies HBeAg+ 8% 19% 26% HBeAg, hepatitis B e antigen; HBsAg, hepatitis B surface antigen. Hou J, et al. N Engl J Med. 2026;394(24):2395-2406. Functional Cure Rates in B-Well 1 and B-W
Slide 36: Synergy of PBGENE-HBV + ASO in HBV Mouse Model
Synergy of PBGENE-HBV + ASO in HBV Mouse Model Drove Deeper Reductions in HBV DNA and HBsAg Improved Depth and Durability of HBV DNA and HBsAg Declines with PBGENE-HBV + ASO HBV DNA HBsAg ASO, antisense oligonucleotide; HBsAg, hepatitis B surface antigen; HBV, hepatitis B virus. Data on file. -60 -5
Slide 37: ELIMINATE-B Trial Moves to Phase 2 Expansion
Establishing PBGENE-HBV as the Foundation of Treatment in HBV: ELIMINATE-B Trial Moves to Phase 2 Expansion PBGENE-HBV 0.4 mg/kg Q8W Viral Cure cccDNA Elimination Endpoint: Undetectable HBV DNA off therapy Functional Cure Suppression of viral infection Endpoint: Undetectable HBV DNA and HBsAg off th
Slide 38: FUNCTION-DMD Clinical Momentum Continues with PBGENE-DMD
FUNCTION-DMD Clinical Momentum Continues with PBGENE-DMD
Slide 39: Phase 1/2 Study Designed for Safety and Speed
Inclusion criteria • Male age 2-7 • DMD with mutations in exons 45-55 • Able to complete age-appropriate motor testing assessments • • Up-to-date vaccines Part 2: Expansion Cohort (n=4-15) Part 1: Initial Safety Cohort (1x1014 vg/kg) (n=3a) Phase 1/2 Study Designed for Safety and Speed Clinical Tria
Slide 40: PBGENE-DMD Continues To Advance In FUNCTION-DMD Study
PBGENE-DMD Continues To Advance In FUNCTION-DMD Study 1st patient successfully dosed August 2026 No serious adverse events, no adverse events of special interest (including no TMA, myocarditis, or acute liver injury) DSMB has reviewed patient 1 data through week 8 No safety concerns identified Endor
Slide 41: Closing Thoughts
Closing Thoughts re
Slide 42: Numerous Upcoming Catalysts in Next 12 Months
Precision BioSciences Has Numerous Upcoming Catalysts in Next 12 Months Key Expected Inflection Points Through 2027 PBGENE- PBGENE-DMD AAV Program 2026 Q4 AASLD Late-Breaking Oral Data Update & Phase 2 Study Plan Safety Update from Initial Patients 2027 H1 Initiate ELIMINATE-B2 Global Ph2 Study in Q
Slide 43: Appendix
Appendix Ha
Slide 44: PBGENE-HBV Positioned to Impact More Patients than Other Gene Therapy Programs
PBGENE-HBV Positioned to Potentially Impact More Patients than Other Gene Therapy Programs 44 AATD, alpha-1 antitrypsin deficiency; ATTRv-CM, hereditary transthyretin amyloidosis with cardiomyopathy; ATTRv-PN, hereditary transthyretin amyloidosis with polyneuropathy; Beta-Thal, beta thalassemia; cHB
Slide 45: 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 cHBV infections globally Up to 2,400,000 cHBV infections in the US Estimated ~5M patients in major markets infected with chronic HBV & treated with standa
Slide 46: Phase 2 Study Endpoints Aligned to Current HBV Regulatory Guidance
Phase 2 Study Endpoints Aligned to Current HBV Regulatory Guidance 46 GUIDANCE DOCUMENT Chronic Hepatitis B Virus Infection: Developing Drugs for Treatment APRIL 2022 New finite duration therapies could be evaluated in clinical trials using any of the following efficacy endpoints: Sustained suppress
Slide 47: ARCUS: Precision's Proprietary Gene Editing Platform
ARCUS: Precision’s Proprietary Gene Editing Platform ARCUS wholly owned by Precision BioSciences Derived from the homing endonuclease I-CreI found in green algae Naturally evolved to drive high-efficiency editing >75 patents issued covering ARCUS and in vivo gene editing Founded in 2006 and dedicate
Slide 48: PBGENE-HBV Directly Eliminates cccDNA; All Other Therapies Suppress
cccDNA Integrated HBV DNA Nucleus HBV transcripts HBsAg HBV DNA HBcrAg HBV RNA PBGENE-HBV Directly Eliminates cccDNA; All Other Therapies Suppress While Preserving cccDNA cccDNA, covalently closed circular DNA; CHB, chronic hepatitis B; HBcrAg, hepatitis B core-related antigen; HBsAg, hepatitis B su
Slide 49: Undetectable pgRNA Associated With Success of Stopping Nucleoside Analogs
Studies Demonstrate Undetectable pgRNA Is Associated With Success of Stopping Nucleoside Analogs and Increasing Probability of Cure cccDNA, covalently closed circular DNA; NA, nucleos(t)ide analog; pgRNA, pregenomic RNA (also known as HBV RNA). 1. Meier MA, et al. J Hepatol. 2021;75(4):840-847. 2. T
Slide 50: ALL EASL Patients Have Achieved 6+ Months of Durable pgRNA Loss
-4 0 4 8 12 16 20 24 28 32 36 40 44 48 52 56 60 Weeks from First Dose Patient 6 0.4 mg/kg Patient 7 0.8 mg/kg Patient 10 0.4 mg/kg Patient 9 0.8 mg/kg Patient 12 0.4 mg/kg NEW AASLD DATA: ALL EASL Patients Have Achieved 6+ Months of Durable pgRNA Loss 50 AASLD, American Association for the Study of
Slide 51: Lower cccDNA and Blood Biomarkers Track with Cure Rates
Lower cccDNA and Blood Biomarkers Track with Cure Rates Lower cccDNA in the liver is associated with significantly better odds of a sustained virologic response (adjusted odds ratio for log cccDNA was 5.3 [95% CI: 1.5-18.2, P = .009]) Sung et al, 2005, Gastroenterology Functionally cured patients sh
Slide 52: HBsAg as a Secondary Biomarker
cccDNA pgRNA HBV DNA (Infectious particles) HBsAg as a Secondary Biomarker Derived From cccDNA and Integrated HBV DNA 52 pgRNA is the cccDNA-specific biomarker HBsAg is a secondary biomarker expected to be reduced with cccDNA elimination cccDNA- specific Biomarker iDNA HBsAg Secondary Biomarker Back
Slide 53: ELIMINATE-B Phase 2 Schema
Establishing PBGENE-HBV as the Foundation of Treatment in HBV: ELIMINATE-B Trial Moves to Phase 2 Expansion Viral Cure cccDNA Elimination Endpoint: Undetectable HBV DNA off therapy Functional Cure Suppression of viral infection Endpoint: Undetectable HBV DNA and HBsAg off therapy Monotherapy Sequent
Slide 54: PBGENE-HBV Lowers Viral Burden and Potential to Increase Functional Cure Rate
Bepirovirsen Most Effective in Patients with Low Viral Burden; PBGENE-HBV Lowers Viral Burden and Potential to Increase Functional Cure Rate PBGENE-HBV Eliminates cccDNA to reduce viral burden Goal of deeper suppression of viral transcripts with Bepirovirsen Potential for a higher functional cure ra
Slide 55: Durable HBsAg Declines
PBGENE- 55 Patients with only 1 dose at 0.4 mg/kg as of the data cutoff have been excluded from this analysis. HBsAg, hepatitis B surface antigen; HBV, hepatitis B virus. Data cutoff: September 28, 2026. Durable HBsAg Declines at -100% -90% -80% -70% -60% -50% -40% -30% -20% -10% 0% 0.40 0.65 0.80 M
Slide 56: PBGENE-HBV Safety Profile with CTCAE
PBGENE-HBV Safety Profile with CTCAE—Planned for Phase 2 CTCAE, Common Terminology Criteria for Adverse Events; AE, adverse event; ALT, alanine aminotransferase; AST, aspartate aminotransferase; DMC, data monitoring committee; HBV, hepatitis B virus; LNP, lipid nanoparticle; SAE, serious adverse eve
Slide 57: Bepirovirsen Does Not Eliminate cccDNA
Bepirovirsen Does Not Eliminate cccDNA, Thus End of Treatment Response is Not Maintained Bepi + NUC Treatment Period NUC-Only Period 24 weeks: End-of-Treatment Response 48 weeks: Assess for Stop NUCs Off-Therapy Period 72 weeks: Primary Outcome 54 29 24 30 32 30 16 39 46 0 20 40 60 80 100 24 weeks 4
Slide 58: PBGENE-DMD
PBGENE-DMD ta
Slide 59: Large Valuation Opportunity for Gene Therapy Drugs in DMD
Large Valuation Opportunity for Transformative Gene Therapy Drugs in DMD This was an increase from ~$3B in late 2017 when it received IND approval to initiate Phase 1 trials for Elevidys After receiving approval for its gene therapy Elevidys for DMD, Sarepta reached a peak market capitalization of ~
Slide 60: PBGENE-DMD Designed to Provide Durable Functional Improvement
PBGENE-DMD Designed to Provide Durable Functional Improvement for 60% of Patients with DMD AAV, adeno-associated virus; DMD, Duchenne muscular dystrophy. 1. Béroud C, et al. Hum Mutat. 2007;28(2):196-202. . 60 PBGENE-DMD: A single AAV encodes 2 ARCUS proteins designed to permanently edit a patient’s
Slide 61: PBGENE-DMD Produces Near-Full-Length Dystrophin Protein
PBGENE-DMD Designed to Produce a Near-Full-Length Dystrophin Protein, Demonstrated to be Functional in Humans ABD, actin-binding domain; CR, cysteine-rich domain; CT, C-terminal domain; DMD, Duchenne muscular dystrophy. Phelps SF, et al. Hum Mol Genet. 1995;4(8):1251-1258. 61 ABD R1 R2 R3 R4 R5 R6 R
Slide 62: Near-Full-Length Dystrophin Has Known Function in Del45-55 Genotype
Near-Full-Length Dystrophin Protein Has Known Function in Individuals with Dystrophin Del45-55 Genotype BMD, Becker muscular dystrophy; DMD, Duchenne muscular dystrophy. 1. Taglia A, et al. Acta Myol. 2015;34(1):9-13. 2. Echigoya Y, et al. J Pers Med. 2018;8(4):41. 3. Béroud C, et al. Hum Mutat. 200
Slide 63: ~5% Dystrophin is Associated with Meaningful Clinical Benefit
~5% Dystrophin is Associated with Meaningful Clinical Benefit 63 ~5% dystrophin associated with prolonged ambulation beyond typical loss of ambulation time (~10–12 years) and linked to improved survival and milder diseases DMD, Duchenne muscular dystrophy. de Feraudy Y, et al. Ann Neurol. 2021;89(2)
Slide 64: PBGENE-DMD Reason-to-Believe: Bridging to Investigation in Humans
PBGENE-DMD Reason-to-Believe: Bridging to Investigation in Humans 64
Slide 65: PBGENE-DMD Increased Dystrophin Protein Levels Over Time
PBGENE-DMD Resulted in Increased Dystrophin Protein Levels Over Time, Exceeding Expected Therapeutic Threshold DMD, Duchenne muscular dystrophy; vg/kg, vector genomes per kilogram. https://investor.precisionbiosciences.com/events/event-details/precision-biosciences-virtual-investor-event-duchenne-mu
Slide 66: Broad Distribution of Dystrophin Positive Cells
Broad Distribution of Dystrophin Positive Cells Across Skeletal, Cardiac, and Respiratory Muscles After PBGENE-DMD Treatment Percentage of cells expressing near-full-length dystrophin protein increases over time after treatment with PBGENE-DMD DMD, Duchenne muscular dystrophy; vg/kg, vector genomes
Slide 67: Dystrophin Restoration in Respiratory Muscles of Juvenile DMD Mice
PBGENE-DMD Achieves Therapeutic Dystrophin Restoration in Critical Respiratory Muscles of Early Juvenile DMD Mice DMD, Duchenne muscular dystrophy; vg/kg, vector genomes per kilogram. https://investor.precisionbiosciences.com/events/event-details/precision-biosciences-virtual-investor-event-duchenne
Slide 68: PBGENE-DMD Improved Muscle Function and Long-Term Durability
PBGENE-DMD Significantly Improved Muscle Function and Demonstrated Long-Term Durability Force was measured in the calf across multiple stimulation frequencies. Averaged force normalized to body weight is shown. Statistically significant (p<0.001) increases in force were observed in both doses of PBG
Slide 69: Transduction in Nonhuman Primates Compared To Mice
PBGENE-DMD Demonstrated as Good as or Better Transduction in Nonhuman Primates Compared To Mice Suggesting Good Translatability AAV, adeno-associated virus; DMD, Duchenne muscular dystrophy; NHP, non-human primate. https://investor.precisionbiosciences.com/events/event-details/precision-biosciences-
Slide 70: Nonclinical Data Support Safety and Efficacy of the Clinical Dose
Nonclinical Data Support the Potential for Safety and Efficacy of the Clinical Dose DMD, Duchenne muscular dystrophy; GLP, good laboratory practice; NHP, non-human primate. 1. Background levels of off-target editing set at 0.2% indels. https://investor.precisionbiosciences.com/events/event-details/p
Slide 71: PBGENE-DMD Investigated at Lower or Equal Doses to Other AAV DMD Programs
PBGENE-DMD to be Investigated at Lower or Equal Doses to All Other AAV DMD Programs Approved or in Development 71 *Discontinued. AAV, adeno-associated virus; DMD, Duchenne muscular dystrophy; GC/kg, genome copies per kilogram; vg/kg, viral genomes per kilogram. 1. Precision BioSciences. PBGENE-DMD.
Slide 72: FUNCTION-DMD Primary and Secondary Endpoints
*Bayley Scales of Infant and Toddler Development are 4th Edition, Bayley-4. AE, adverse event; DMD, Duchenne muscular dystrophy; DMO, digital mobility outcome; NSAA, North Star Ambulatory Assessment; SAE, serious adverse event; TRAE, treatment-related adverse event. Precision BioSciences. FUNCTION-D
Slide 73: Key Inclusion Criteria
AAV, adeno-associated virus; DMD, Duchenne muscular dystrophy; NSAA, North Star Ambulatory Assessment. Updated February 24, 2026. https://clinicaltrials.gov/study/NCT07429240. Accessed February 25, 2026. 73 Category Inclusion Criteria Age / Sex Males, 2-7 years of age (inclusive) at time of consent
Slide 74: Safety as Top Priority: Immune Modulation Regimen Strategy
Safety as Top Priority: Immune Modulation Regimen (IMR) strategy ensures monitoring and interventions are optimized to patient safety needs during 2 key safety risk periods Early immune and complement activation can cause blood count changes, cardiac strain, inflammation, and flu-like symptoms. AAV,