Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — Corporate Update (May 2025)
38 slides from Precision Biosciences's corporate update, May 2025
Slide 1: Precision BioSciences Muscle Programs Update
Precision BioSciences Muscle Programs Update May 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: Opening Remarks and Overview
Opening Remarks and Overview Michael Amoroso President & Chief Executive Officer Precision BioSciences
Slide 4: Precision BioSciences Continues to Validate ARCUS Clinically Through 2025
Precision BioSciences Continues to Validate ARCUS Clinically Through 2025 Through our clinical programs, over 100 patients have received ARCUS-based therapies safely for hematological malignancies, chronic hepatitis B, and OTC deficiency. Wholly-owned PBGENE-HBV ECUR-506 partnered program demonstrat
Slide 5: Precision Biosciences
PRECISION BIOSCIENCES Founded in 2006 and dedicated to developing novel therapeutics designed to overcome and potentially cure difficult-to-treat diseases with high unmet need, including infectious and rare genetic diseases. Precision’s therapies are developed utilizing ARCUS, a proprietary next gen
Slide 6: PBGENE-DMD is 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 6
Slide 7: 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 7
Slide 8: Today, 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 9: Clear Regulatory Guidance on Clinical Development Path to Approval
Clear Regulatory Guidance on Clinical Development Path to Approval Validated Clinical Trial Design with Potential for Accelerated Approval 〉 Pivotal design and accelerated pathway aligned with the FDA in DMD space Phase I/II Phase III Dose Evaluation Expansion Cohort Pivotal Study Evaluate dose leve
Slide 10: Global DMD Market Poised for Innovative Breakthroughs Like PBGENE-DMD
Global DMD Market Poised for Innovative Breakthroughs Like PBGENE-DMD Prevalence & Incidence 1 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 ba
Slide 11: PBGENE-DMD Therapeutic Approach
PBGENE-DMD Therapeutic Approach Cassie Gorsuch, PhD Chief Scientific Officer Precision BioSciences, Inc
Slide 12: DMD is a Genetic Disorder Resulting in Progressive Muscle Degeneration and Early Death
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 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 Healthy DMD 1 2 3 4 5 6 7 8 9 10 11 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 12 13 14 15 16 17 18 19 20 21 22 23 24 25 26 27 28 29 30 31 32 33 23 24 25 26 27 28 29 30 31 32 33 34 35 36 37
Slide 14: PBGENE-DMD Designed to Provide Durable Functional Improvement for Majority Patients with DMD
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 15: ARCUS is Uniquely Positioned for DMD Gene Editing
ARCUS is Uniquely Positioned for DMD Gene Editing Why Other Gene Editors Can Not Follow Unique Smallest Single Cut gene editor component editor Promotes safety Enables delivery Increases efficiency DMD: Safety and Efficacy DMD: Safety and Efficacy DMD: Efficacy Predictable repair enhances Both nucle
Slide 16: PBGENE-DMD is 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 16
Slide 17: PBGENE-DMD Showed Durable Functional Improvement Over Time
PBGENE-DMD Showed Durable Functional Improvement Over Time
Slide 18: PBGENE-DMD Significantly Improved Muscle Function and Demonstrated Long-Term 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 to Untreated Disease 150% 100% 50% 0% 〉Improved muscle function observed from 3 to 6 mont
Slide 19: Long-term Functional Improvement Driven by Increasing and Stable Levels of Dystrophin
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 20: PBGENE-DMD Restored Functional Dystrophin Protein in Majority of Myofibers in Calf
PBGENE-DMD Restored Functional Dystrophin Protein in Majority of Myofibers in Calf PBGENE-DMD Untreated Untreated Disease model Disease model Healthy control model Dystrophin Laminin Broad and substantial near full-length functional dystrophin restoration across muscles ASGCT 2025. 20
Slide 21: Editing Satellite Cells is Essential for Durable Effect
Editing Satellite Cells is Essential for Durable Effect Satellite cells are the resident stem cells in skeletal muscle and Myocytes Myofiber essential for muscle regeneration In DMD where myofiber degeneration is continuous, editing satellite cells is essential for long-term therapeutic effect 21
Slide 22: 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 23: Novel Mechanism and Proven Dystrophin Protein
Novel Mechanism and Proven Dystrophin Protein
Slide 24: PBGENE-DMD's Novel Mechanism Enables Durable Improvements Independent of AAV Persistence
PBGENE-DMD’s Novel Mechanism Potentially Enables Durable Improvements in Muscle Function Independent of the Persistence of AAV PBGENE-DMD: The dystrophin protein is expressed by the human genome, preventing the need for AAV persistence Microdystrophin Gene Therapies: Microdystrophin protein is expre
Slide 25: PBGENE-DMD Designed to Produce a 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 26: PBGENE-DMD Designed to Produce a 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 27: Long-term Functional Improvement Driven by Increasing and Stable Levels of Dystrophin
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 28: Near Full Length Dystrophin Protein 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 29: PBGENE-DMD Designed With Safety Top Of Mind
PBGENE-DMD Designed With Safety Top Of Mind
Slide 30: PBGENE-DMD Designed For Safety Through Low Dose and High Quality AAV
PBGENE-DMD Designed For Safety Through Low Dose and High Quality AAV Permanently correct the gene using just enough AAV Because PBGENE-DMD corrects the DNA, AAV persistence is not necessary, and therefore lowest efficacious dose is the goal Consistently produce high quality AAV Precision BioSciences
Slide 31: PBGENE-DMD Significantly Improved Muscle Function at even the Lowest Dose Level
New Data PBGENE-DMD Significantly Improved Muscle Function at even the Lowest Dose Level Untreated Untreated PBGENE-DMD PBGENE-DMD Diseased Healthy (1e14 vg/kg) (3e13 vg/kg) 200% Normalized Force/BW 150% to Untreated Disease 100% 50% 0% Significant Improvement in Muscle Force Even with the Lowest Do
Slide 32: 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 33: KOL Panel Discussion
KOL Panel Discussion Russell Butterfield, MD, PhD Cassie Gorsuch, PhD Director, Center for Gene Therapy Chief Scientific Officer Associate Professor, Child Neurology Precision BioSciences, Inc University of Utah, School of Medicine 33
Slide 34: Program Next Steps
Program Next Steps Including Implications to the PBGENE-3243 Program 34
Slide 35: 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 35
Slide 36: PBGENE-3243 Program Development Paused
PBGENE-3243 Program Development Paused —Exploring Opportunities for Advancement Precision BioSciences remains We are encouraged by the We are grateful to the committed to our goal of preclinical data supporting mitochondrial community continuing to advance ARCUS- the potential for PBGENE-3243 to inc
Slide 37: Precision BioSciences is a 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 38: Q&A
Q&A re 38