Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — Investor Presentation (September 2025)
44 slides from Precision Biosciences's investor presentation, September 2025
Slide 1: Corporate Deck September 2025
Corporate Deck September 2025
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements, as may any related presentations, within the meaning of the Private Securities Litigation Reform Act of 1995. The Company intends such forward-looking statements to be covered by the safe harbor provisions for forward-
Slide 3: Leadership Team Brings Strategic and Operational Experience
Precision BioSciences Leadership Team Brings Strategic and Operational Experience Michael Amoroso Jeff Smith, Ph.D. Cassie Gorsuch, Ph.D. Alex Kelly Cindy Atwell Since Oct 2021 Since March 2006 Since June 2016 Since Oct 2020 Since April 2019 President & Chief Research Officer, Chief Scientific Offic
Slide 4: Clinical Stage In Vivo Gene Editing Company
Precision BioSciences is a Clinical Stage In Vivo Gene Editing Company Expected First Clinical Multiple Programs in or Nearing Clinical Data Data Timing 〉 Investigational New Drug (IND) clearance and Fast Track designation PBGENE-HBV received in United States 2025 In Vivo Gene Editing 〉 Global phase
Slide 5: Gene Editing Platform Best Suited for Our Indications
We Believe our Gene Editing Platform is Best Suited for the Indications We are Pursuing
Slide 6: ARCUS is Our Proprietary Gene Editing Platform
ARCUS is Our Proprietary Gene Editing Platform: Naturally Evolved to Drive High Efficiency Editing • ARCUS wholly owned by Precision BioSciences • Evolved to safely edit by inserting in genome, adding function CRISPR-based editing tools engineered from enzymes evolved ARCUS to knockout DNA only •
Slide 7: The ARCUS Cut is Uniquely Designed to Drive Defined Outcomes
The ARCUS Cut is Uniquely Designed to Drive Defined Outcomes ARCUS cut leads to HDR or “Perfect” Re-ligation Cas9 C C G TAT G C A AT TA G C TA G G T C G Blunt cut G G C ATA C G T TA AT C G AT C C A G C induces NHEJ The ARCUS Cut TALEN/ZFN Generates a DNA replication direction C C G TAT G C A AT TA G
Slide 8: Size Matters for Where You Can Deliver
Size Matters for Where You Can Deliver ARCUS can use different delivery vehicles to target diverse tissue types ARCUS LNP AAV Liver Eye Muscle CNS Other 7
Slide 9: Size Matters for What You Can Deliver
ARCUS 1500 bp SaCas9 4200 bp AAV Genome Capacity ~4700 bp Size Matters for ARCUS Cas12a 4900 bp What You Can Deliver: Small ARCUS Size Allows SpCas9 Two Nucleases in One AAV 5100 bp ARCUS for Gene Excision as in our PBGENE-DMD program Base Editor (ABEmax) 5800 bp – 6500 bp Prime Editor (PEmax) 7200
Slide 10: Simplicity: ARCUS is the Only Single Protein Component Editor
Simplicity: ARCUS is the Only Single Protein Component Editor ARCUS Single protein with a DNA recognition motif and catalytic 1 activity all in one; no guide RNA required Editing outcome not dependent on simultaneous delivery of multiple components leading to higher efficiency Single component requi
Slide 11: ARCUS for the More Sophisticated Gene Edit
ARCUS for the More Sophisticated Gene Edit Designed by nature for a multitude of applications versus other gene editing modalities Replace Remove defective and insert functional gene Insertion Insert gene to add function Degree of Difficulty ARCUS Remove portion of genome, high Capability To Perform
Slide 12: OTC Deficiency Program: First ARCUS in vivo Program Validated In Clinic
Ornithine Transcarbamylase (OTC) Deficiency Program: Partnered Program First ARCUS in vivo Gene Editing Program Validated In Clinical Study Employing ARCUS Nuclease Initial Clinical Validation for ARCUS in vivo Gene Insertion First Infant Achieves Complete Clinical Response in Our Partner iECURE’s P
Slide 13: PBGENE-HBV is First Wholly Owned In Vivo Gene Editing Application
PBGENE-HBV is First Wholly Owned In Vivo Gene Editing Application of ARCUS
Slide 14: 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 15: Chronic Hepatitis B Program Validated In Clinical Study
Chronic Hepatitis B Program Validated In Clinical Study: Phase 1 Safety and Efficacy Established in Cohort 1 with Proof-of-Activity and Well-Tolerated Profile Observed in All Patients • Investigational New Drug Application cleared in the U.S. and Clinical Trial Applications approved in Moldova, Hong
Slide 16: Risk of Liver Cancer, Complications, and Mortality Remain on Current SoC
Risk of Liver Cancer, Complications, and Mortality Remain on Current SoC: cHBV Patients Have Up to a 30% Risk of Liver Cancer Over 10 years on Nucleos(t)ide Analogs Up to 40% of patients with chronic HBV infections may develop life-threatening complications including cirrhosis and/or HCC1 cHBV Resul
Slide 17: cccDNA and Integrated HBV DNA are the Viral Source of Downstream Markers
cccDNA and Integrated HBV DNA are the Viral Source of Downstream Markers Viral Source cccDNA Integrated HBV DNA Hepatocytes Bloodstream HBsAg mRNAs pgRNA Downstream HBV RNA Markers HBcrAg HBeAg HBsAg Infectious virions (HBV DNA) cccDNA, covalently closed circular DNA; DNA, deoxyribonucleic acid; HBe
Slide 18: 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 HBV PBGENE-HBV is uniquely designed to seek a complete Viral cure by eliminating cccDNA and Source 1 cccDNA cccDNA Integrated HBV2DNA inactivating integrated HB
Slide 19: Current Strategies Rarely Achieve Functional Cure Because They Leave cccDNA Present
Current Therapeutic Strategies Rarely Achieve Functional Cure Because They Leave cccDNA Present cccDNA Integrated HBV DNA Hepatocytes Bloodstream D Current therapies in HBsAg mRNAs pgRNA C development target HBV RNA downstream Even combining antivirals and A B immune modulators, functional component
Slide 20: ≥30% Functional Cure Rate Set By HBV Experts Has Remained Elusive
≥30% Functional Cure Rate Set By HBV Experts Has Remained Elusive PBGENE-HBV has opportunity to be clinically meaningful by increasing functional cures Current Approved Drug Modalities or in Late-stage Development Clinically Meaningful show single-digit functional cure rates Average Functional Cure
Slide 21: Phase 1/2a ELIMINATE-B Trial Overview
5 Countries HBeAg-negative patients controlled on nucleos(t)ide analogs Up to 45 Patients ClinicalTrials.gov identifier NCT06680232. 20
Slide 22: Phase 1/2a Study Design
Phase 1/2a Study Design Potential for rapid evaluation of safety and efficacy of PBGENE-HBV in 2025 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 com
Slide 23: Phase 1/2a Study Goals to Demonstrate Both Safety and Efficacy
Phase 1/2a Study Goals to Demonstrate Both Safety and Efficacy Key Endpoints Safety determined by: Efficacy determined by: Frequency and severity of dose-limiting Antiviral activity through fixed duration toxicities (DLTs) PBGENE-HBV treatment Monitoring Biomarkers Reduction/Negativity in HBsAg: Sus
Slide 24: Status - Completed Dosing Cohort 1, Cohort 2 Partially Complete
Status - Completed Dosing Cohort 1, Cohort 2 is Partially Complete and Cohort 3 is Underway DATA CUT: We have now successfully enrolled 6 evaluable patients into cohort 1 (n=3) and cohort 2 (n=3) with full analysis Safety Efficacy 〉In Cohort 1 at 0.2 mg/kg no adverse events 〉Proof-of-activity establ
Slide 25: Sustained HBsAg Reduction Seen in 1 of 3 Patients at Lowest Dose
Dose Level 1 | 0.2 mg/kg EFFICACY: Sustained HBsAg Reduction Seen in 1 of 3 Patients at Lowest Dose Level August 6, 2025 Company Press Release 24
Slide 26: PBGENE-DMD
PBGENE-DMD Excision of a section of the Dystrophin Gene responsible for ~60% of the defects in Duchenne’s Muscular Dystrophy (DMD)
Slide 27: PBGENE-DMD Opportunity
PBGENE-DMD Opportunity: Potential to provide a first-in-class and best-in-class therapeutic for patients High Unmet Need Clear Regulatory Growing Opportunity for Guidance Innovative Treatments 26
Slide 28: DMD is a Genetic Disorder Resulting in Progressive Muscle Degeneration
DMD is a Genetic Disorder Resulting in Progressive Muscle Degeneration and Early Death Brain 〉Learning disability or serious cognitive disability in a subset of patients Heart 〉Cardiomyopathy and arrythmias 〉Eventual cardiac failure leading to premature death in late teens or 20s Diaphragm & Interco
Slide 29: Patients with DMD are in Dire Need with Limited Therapeutic Options
Today, Patients with DMD are in Dire Need with Limited Therapeutic Options Ideal therapy Microdystrophin Exon Skipping PBGENE-DMD would have: Gene Therapies Therapies Target Product Profile Improved muscle function over time Long-term durable benefit Broadly applicable to patients Corrects human dys
Slide 30: 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 ~20% growth $8B DMD Patients globally DMD Patients in US rate >20k births ~550 births per year globally per year in US 2025 2030 1. Prevalence and Incidence bas
Slide 31: PBGENE-DMD Designed to Provide Durable Functional Muscle Improvement
Novel Mechanism Corrects Human Dystrophin Gene Resulting in a Functional Dystrophin Protein PBGENE-DMD is Designed to Provide Durable Functional Muscle Improvement for the Majority of Patients with DMD Designed For Safety Through Lower Dose AAV 30
Slide 32: PBGENE-DMD Designed to Provide Durable Functional Improvement
PBGENE-DMD Designed to Provide Durable Functional Improvement for Majority 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 ARCUS 1 ARCUS 2 ARCUS 1 Dystrophin gene
Slide 33: ARCUS is Uniquely Positioned for DMD Gene Editing
ARCUS is Uniquely Positioned for DMD Gene Editing Why Other Gene Editors Cannot Follow Unique Smallest Single Cut gene editor component editor Promotes safety Enables delivery Increases efficiency DMD: Potential Safety DMD: Potential Safety DMD: Potential Efficacy and Efficacy and Efficacy Iterative
Slide 34: PBGENE-DMD Showed Durable Functional Improvement Over Time
PBGENE-DMD Showed Durable Functional Improvement Over Time
Slide 35: PBGENE-DMD Significantly Improved Muscle Function and Durability
New Data 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 mont
Slide 36: Long-term Functional Improvement Driven by Increasing Dystrophin Expression
New Data Long-term Functional Improvement Driven by Increasing and Stable Levels of Dystrophin Protein Expression Heart Calf Quadricep 20 30 25 25 20 Dystrophin Restoration (%) Dystrophin Restoration (%) Dystrophin Restoration (%) 15 20 15 10 15 10 10 5 5 5 0 0 0 Treated disease mice Treated disease
Slide 37: PBGENE-DMD Edited Muscle Satellite Stem Cells
PBGENE-DMD Edited Muscle Satellite Stem Cells, Providing Potential for Durable Functional Improvement Edited PAX7+ cell PBGENE-DMD has demonstrated permanent editing of satellite cells, beyond transient transduction Observed edited dystrophin mRNA in PAX7+ cells, a marker for muscle satellite stem c
Slide 38: Novel Mechanism and Proven Dystrophin Protein
Novel Mechanism and Proven Dystrophin Protein
Slide 39: PBGENE-DMD Designed to Produce Near Full-Length Dystrophin Protein
PBGENE-DMD Designed to Produce a Near Full-Length Dystrophin Protein, Proven 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 “ H1 H2 H4 ABD R1 R2 R3 R24 CR Truncated
Slide 40: PBGENE-DMD Designed to Produce Near Full-Length Dystrophin Protein
PBGENE-DMD Designed to Produce a Near Full-Length Dystrophin Protein, Proven 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 H1 H2 H4 Functional ABD R1 R2 R3 R4 R5 R6
Slide 41: Near Full-Length Dystrophin Has Proven Function in Del45-55 Genotype
Near Full-Length Dystrophin Protein Has Proven 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 • Asymptomatic or mild leading to loss
Slide 42: PBGENE-DMD Program is Quickly Moving Towards Clinical Stage
PBGENE-DMD Program is Quickly Moving Towards Clinical Stage Identified Clinical Candidate Received Positive Pre-IND Feedback From FDA Initiated GLP Toxicology Studies Manufacture Clinical Trial Material Target Filing of CTA and/or IND in 2025 Initiate First-in-Human Clinical Study 41
Slide 43: PBGENE-DMD: Highly Desirable Target Product Profile
PBGENE-DMD: Highly Desirable Target Product Profile Ideal therapy PBGENE-DMD Potential to Provide Best-In-Class Therapeutic Profile would have: Improvements in muscle Nonclinical evidence of muscle function improvement over time function Long-term durable benefit Data demonstrating improvement is du
Slide 44: Meaningful Catalysts in Next 6-18 Months; Cash Runway Into 2H 2027
Precision BioSciences Offering Meaningful Catalysts in Next 6-18 Months Ample cash runway to fund regulatory submissions and clinical data readouts CASH RUNWAY INTO 2H 2027 ~$85M Potential to Advance 3 Programs to Phase I Clinical Data Cash and cash equivelents1 As of 6/30/25 Expected Inflection Poi
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