Slide 1: PBGENE-DMD Investor Update
Precision BioSciences PBGENE-DMD Investor Update March 17, 2026
Slide 2: Forward-Looking Statements
Forward-Looking Statements 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 statements,
Slide 3: Opening Remarks and Overview
Opening Remarks and Overview Alex Kelly Chief Financial Officer Precision BioSciences 3
Slide 4: ARCUS: Precision's Proprietary Gene Editing Platform
ARCUS: Precision’s Proprietary Founded in 2006 and dedicated Gene Editing Platform to developing novel therapeutics designed with 〉 ARCUS wholly owned by Precision BioSciences goal of curing difficult-to-treat 〉 Derived from the homing endonuclease I-CreI found in green algae diseases with high unme
Slide 5: ARCUS Platform Has Multiple Points of Clinical Validation
ARCUS WORKS: ARCUS Platform Has Multiple Points of Clinical Validation Led by Wholly Owned HBV Program Clinical Stage Programs Validating ARCUS Wholly Owned Programs PBGENE-HBV for Chronic Hepatitis B PBGENE-DMD for Duchenne Muscular Dystrophy 〉 Viral gene elimination program with over 30 doses deli
Slide 6: The Focus for Today: PBGENE-DMD
The Focus for Today is Our Second Wholly Owned Clinical-Stage Program: PBGENE-DMD Cassie Gorsuch, PhD Chief Scientific Officer Precision BioSciences 6
Slide 7: PBGENE-DMD: designed to excise the exons 45-55 hot-spot region
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 7 7
Slide 8: Approach Has Potential to Surpass 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 9: Reasons to Believe in FUNCTION-DMD Phase 1/2 Trial
Reasons to Believe in FUNCTION-DMD Phase 1/2 Trial: Potential to provide a first-in-class and best-in-class therapeutic for patients Unmet ARCUS Robust Preclinical Clinical Trial Designed World Class DMD Need Persists Gene editing Data for Safety Clinical Trial Sites Patients need better Addressing
Slide 10: Global DMD Market Poised for Innovative Breakthroughs
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 11: The Unmet Need Persists Despite Advancements in DMD Therapeutic Development
The Unmet Need Persists Despite Advancements in DMD Therapeutic Development Pat Furlong President & Founder Parent Project Muscular Dystrophy (PPMD) 11
Slide 12: DMD is a Progressive Neuromuscular Disorder Leading to Early Mortality
DMD is a Progressive Neuromuscular Disorder Leading to Early Mortality 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 • From birth: Damage starts right • Muscle weakness often becomes • Walking gets tougher • Full-time wheelchair use is • In late-stage disease, away, but many boys look and act
Slide 13: 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 14: The Unmet Needs Persists: Ideal therapy allows early intervention
The Unmet Needs Persists: The ideal therapy would allow for early intervention to promote increased muscle function 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 Despite advancements in DMD therapies, the unmet need persists and continued investment in novel approaches like PBGENE-DMD are ess
Slide 15: PBGENE-DMD Reason-to-Believe: Novel Therapeutic Approach
PBGENE-DMD Reason-to-Believe: Novel Therapeutic Approach Cassie Gorsuch, PhD Chief Scientific Officer Precision BioSciences 15
Slide 16: 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 17: PBGENE-DMD Looks to Improve Upon Existing Therapies for DMD
PBGENE-DMD Looks to Improve Upon Existing Therapies for DMD Broadly applicable Improved Long-term, Near-full length Single to most DMD muscle function durable benefit dystrophin protein, administration patients over time known to function therapy in humans DMD, Duchenne muscular dystrophy. 17
Slide 18: 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 19: Near Full-Length Dystrophin Has Known Function in Del45-55 Individuals
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 20: ~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 21: Durable Improvements Independent of Persistence of AAV Transgene
PBGENE-DMD is Designed for Durable Improvements in Muscle Function Independent of the Persistence of AAV Transgene Microdystrophin Gene Therapies: PBGENE-DMD: Microdystrophin protein is expressed from the AAV The dystrophin protein is expressed by the human transgene, requiring persistence of the AA
Slide 22: PBGENE-DMD Reason-to-Believe: Robust Preclinical Data
PBGENE-DMD Reason-to-Believe: Robust Preclinical Data 22 22
Slide 23: Comprehensive Preclinical Data to Support Safety and Efficacy
Comprehensive Preclinical Data to Support PBGENE-DMD Safety and Efficacy ARCUS overhang cut promotes Durable muscle function high frequency and fidelity excision for improvements in DMD mice predictable repair outcomes out to 9 months post dosing Well-tolerated for Increased dystrophin protein and ≥
Slide 24: Drives Durable Dystrophin Expression and Muscle Function in DMD Mice
PBGENE-DMD Drives Durable Dystrophin Protein Expression, Improvements in Muscle Integrity, and Significant Increases in Muscle Function in DMD Mice Re-establishes Indicates Translates into structural stability healthier muscle increased of muscle fibers tissue following muscle force treatment output
Slide 25: PBGENE-DMD Reason-to-Believe: Bridging to Investigation in Humans
PBGENE-DMD Reason-to-Believe: Bridging to Investigation in Humans 25 25
Slide 26: Significantly Improved Muscle Function and Long-Term Durability
PBGENE-DMD Significantly Improved Muscle Function and Demonstrated 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 months. 〉Dura
Slide 27: As Good or Better Transduction in Nonhuman Primates Than Mice
PBGENE-DMD Demonstrated As Good or Better Transduction in Nonhuman Primates Compared To Mice Suggesting Good Translatability Quad Gastroc NHP Diaphragm Mouse Heart 0 50 100 150 200 250 300 AAV Transduction (% of mouse) Strong Rationale for Go-Forward Dose Advancing in Humans AAV, adeno-associated vi
Slide 28: Nonclinical Data Supports Safety and Efficacy of the Clinical Dose
Nonclinical Data Supports the Potential for Safety and Efficacy of the Clinical Dose SAFETY EFFICACY Evaluated in DMD mouse model and NHPs Evaluated in humanized DMD mouse model 〉 No adverse safety findings observed at the 〉 Similar levels of dystrophin protein restoration completion of two GLP stud
Slide 29: High Quality AAV to Support Clinical Study
PBGENE-DMD Reason-to-Believe: High Quality AAV to Support Clinical Study 2929
Slide 30: Manufacturing Process Produced High Quality AAV With >90% Full Capsids
Manufacturing Process Produced High Quality AAV With >90% Full Capsids 90% Full Capsid AAV Batch 50% Full Capsid AAV Batch Full Capsids Empty Capsids Both full and empty capsids are present in batches of AAV. The ratio of the two is an important quality attribute for safety and efficacy of AAV produ
Slide 31: Minimizing Empty Capsids, Reducing Total Capsids Dosed
Why does this Matter? PBGENE-DMD Aims to Minimizes Empty Capsids, Reducing Total Capsids Dosed and Potentially Improving Safety Full Capsids Empty Capsids PBGENE-DMD Full capsid dose: 1 x 1014 vg/kg 1.1 x 1014 vg/kg Example of 50% Full Ratio Full capsid dose: AAV-based 1 x 1014 vg/kg 2 x 1014 vg/kg
Slide 32: FUNCTION-DMD Study
FUNCTION-DMD Study Aravindhan Veerapandiyan, M.D. Director, Comprehensive Neuromuscular Program, Arkansas Children’s Hospital, University of Arkansas for Medical Sciences 32
Slide 33: What to Expect in DMD Patients?
What to Expect in DMD patients? Underlying disease biology drives several expected baseline findings in young DMD patients 〉 Falls and gait abnormalities are common disease manifestations — Frequent falls, toe walking, and mobility challenges occur as part of the natural history of Duchenne 〉 Develo
Slide 34: 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 35: 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 Company Therapy Vector Dose level(s) tested PBGENE-DMD1 AAV9 1 × 10¹⁴ vg/kg SGT-0012* AAV9 Up to 2 × 10¹⁴ vg/kg SGT-0033 AAV9-derived 1 × 10¹⁴ vg/kg fordadistrogene movaparvovec AAV9 Up to
Slide 36: 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 37: 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 38: 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 two key safety risk periods Safety Mitigation: Preventing early and late serious immune complications. AAV therapies can trigger two predictable immune-r
Slide 39: 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 40: Closing Remarks & Program Next Steps
Closing Remarks & Program Next Steps Alex Kelly Chief Financial Officer Precision BioSciences 40
Slide 41: Upcoming 2026 Milestones and Outlook
Upcoming 2026 Milestones and Outlook PBGENE-HBV PBGENE-DMD • Exploring multiple levers (dose level, number of dose • US IND clearance by FDA in early Q1 2026 administration & time between dose administrations) — IRB process and site activation underway to optimize therapeutic index following IND cle
Slide 42: Q&A
Q&A “a
Slide 43: Appendix Slides
Appendix Slides my
Slide 44: Significantly Improved Muscle Function and Long-Term Durability
PBGENE-DMD Significantly Improved Muscle Function and Demonstrated 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 months. 〉Dura
Slide 45: Drives Durable Dystrophin Expression and Muscle Function in DMD Mice
PBGENE-DMD Drives Durable Dystrophin Protein Expression, Improvements in Muscle Integrity, and Significant Increases in Muscle Function in DMD Mice Dystrophin Protein Muscle Integrity Muscle Function Restoration Biomarkers Improvements Gastrocnemius Creatine Kinase 30 200% Average Force Output2 2500