Precision BioSciences, Inc. · NASDAQ:DTIL
Precision BioSciences — Investor Presentation (August 2023)
36 slides from Precision BioSciences's investor presentation, August 2023
Slide 1: Precision BioSciences Corporate Presentation
Precision BioSciences Corporate Presentation August 2023
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. All statements contained in this herein and in any related presentation that do not relate to matters of his
Slide 3: A Clinical Stage Gene Editing Company Built on Wholly Owned ARCUS Platform
Precision BioSciences: A Clinical Stage Gene Editing Company Built on Wholly Owned ARCUS Genome Editing Platform ARCUS® Platform for therapeutic drug development Pioneers in genome editing technology development ARCUS platform and nucleases are unencumbered by third-party IP; control of over 100 In
Slide 4: 2022 Accomplishments
2022 Accomplishments Presented compelling clinical allogeneic CAR T data to date; 100% ORR 73% CR1 Secured $75M for in vivo gene editing collaboration with Novartis for hemoglobinopathies Raised $50M in equity financing from new and existing investors Extended the cash runway; Runway greater than 2
Slide 5: Differentiating the ARCUS platform
Differentiating the ARCUS platform Highly differentiated genome editing platform for high unmet needs in genetic diseases
Slide 6: Three Distinct Advantages of ARCUS Genome Editing Platform
ARCUS Three Distinct Advantages of ARCUS Genome Editing Platform ARCUS® Platform • ARCUS is inactive until it binds to target DNA Precision for Therapeutic• Drug Development Off-target editing easier to detect and eliminate (Safety & Specificity) • Iterative nuclease optimization process to customiz
Slide 7: ARCUS: Engineering Nucleases that Mimic Nature's Genome Editing System
ARCUS ARCUS: Engineering Nucleases that Mimic Nature’s Genome Editing System The ARCUS nuclease is the only gene editor derived from a natural homing endonuclease Active state ARCUS Inactive state ARCUS Cuts with 3’ overhang that results in a unique jigsaw like pattern The ARCUS nuclease cuts DNA at
Slide 8: ARCUS is Capable of Simple Edits and Optimally Suited for Complex Edits
ARCUS ARCUS is Capable of Simple Edits and Optimally Suited for Complex Edits A deletion (also known as knock-out) is when a gene is made Simple Deletion inoperative through a permanent change in the DNA; therefore, Edit the gene no longer expresses a functional protein An insertion (also known as k
Slide 9: Precise 3' Overhang Cuts are Unique
ARCUS Precise 3’ Overhang Cuts are Unique - Designed to Enable Gene Insertion and Complex Edits and Provide Identifiable Signature for On-Target Editing Complex/Homology-directed Repair (HDR) Simple/Non-Homologous End Joining (NHEJ) ARCUS CRISPR/CAS GENE Cut with 3’ Blunt cut overhang GE NE N Repair
Slide 10: Effective Gene Editing Requires Both AAV and LNP Delivery Capabilities
ARCUS Effective Gene Editing Requires Both AAV and LNP Delivery Capabilities ARCUS can be effectively delivered with both AAV and LNP AAV LNP-mRNA • Sustained nuclease expression may provide increased • Potential to repeat dose to maximize therapeutic impact PROS efficacy PROS • Scalability for manu
Slide 11: Unlocking Greater In Vivo Editing Potential via Safe Harbor Gene Insertion
ARCUS Unlocking Greater In Vivo Editing Potential via Safe Harbor, ARCUS Gene Insertion Inactive state Active state A “safe harbor” ARCUS ARCUS ARCUS nuclease may be used Safe harbor to develop multiple products to insert a different gene for each disease Complex Edit Healthy gene Insertion Edited g
Slide 12: In Vivo Gene Editing Programs
In Vivo Gene Editing Programs Precision in vivo gene insertion and complex gene editing programs highlight unique ARCUS attributes
Slide 13: Broad and Deep In Vivo Pipeline Showcases ARCUS
In Vivo Broad and Deep In Vivo Pipeline Showcases ARCUS for Gene Insertion and Complex Edits PROGRAM INDICATION TISSUE TARGET COMPLEX EDIT RESEARCH CANDIDATE IND- PARTNER TYPE / DELIVERY SELECTION ENABLING PBGENE-HBV Chronic hepatitis B Liver HBV Deletion/LNP Sickle cell disease/ PBGENE-HbE HSCs — I
Slide 14: ARCUS Safety Supported by Long-term Non-Human Primate Data
ARCUS Safety Supported by Long-term Non-Human Primate Data Differentiated ability to track and minimize off-target editing
Slide 15: Five Years of Follow-up for ARCUS in NHP
In Vivo Five Years of Follow-up for ARCUS in NHP; Longest, Publicly Known Gene Editing Data Non-Human Primate Animal Model One-time delivery of an AAV-ARCUS vector into PCSK9 safe harbor for deletion resulted in long-term reductions of serum PCSK9 and LDL1 (5-year timepoint approaching) with no addi
Slide 16: ARCUS Safety and Specificity for Tracking Off-Target Editing
In Vivo ARCUS Safety and Specificity for Tracking Off-Target Editing Oligo Capture: A genome-wide assay for ARCUS off-target editing ARCUS Step 1: Transfect cells with ARCUS DNA “tag” and a DNA “tag” + + AGCT TCGA Step 2: The “tag” is captured at DNA breaks TCGA AGCT resulting from ARCUS cleavage 5’
Slide 17: ARCUS for Gene Insertion
ARCUS for Gene Insertion Wholly owned and partnered programs, including those for large gene insertions
Slide 18: Hemophilia B Gene Insertion: Stable Transduction of Factor IX in Macaque
In Vivo Hemophilia B Gene Insertion: Stable Transduction of Factor IX in Newborn and Infant Macaque Liver (Non-Human Primates)1 Newborn 3-mo infant 6-mo infant 3-month biopsy 1-year biopsy 3-month biopsy 1-year biopsy 3-month biopsy 20-196 (d84) 20-196 (d366) AH0120 (d98) AH0120 (d364) 21-120 (d100)
Slide 19: OTC Clinical Candidate Shows Stable Gene Insertion at Year 1
In Vivo Ornithine Transcarbamylase Deficiency (OTC) Clinical Candidate Shows Stable Gene Insertion Gene Insertion at Year 11 for OTC > Delivery of twin AAV-based vectors carrying ARCUS nuclease Long-term stability of the edited genome vector (GTP-506A) and therapeutic donor vector (GTP- in NHPs was
Slide 20: Gene Insertion to a Safe Harbor Locus in Hematopoietic Stem Cells
In Vivo Gene Insertion to a “Safe Harbor” Locus in Hematopoietic Stem Cells Gene Insertion for SCD and Beta Thal > Permanent integration of an antisickling gene into a ARCUS will be used to add an antisickling gene to “safe harbor” locus in HSCs is expected to prevent the hematopoietic stem cells (H
Slide 21: ARCUS for Complex Edits
ARCUS for Complex Edits 〉HBV approach edits DNA at two locations 〉DMD approach makes a 500,000 base pair edit
Slide 22: Precision Approach in HBV: Excision in Two Locations in the Liver
In Vivo Precision Approach in HBV: Excision in Two Locations in the Liver Gene Excision for Curing HBV requires targeting two key viral components – cccDNA and integrated HBV DNA Chronic HepB > 1. cccDNA CU S Degradation by A R exonuclease cccDNA is a pool of minichromosomes HBV DNA break HBV that h
Slide 23: ARCUS HBV Program Efficacy Data from In Vivo POC Studies
In Vivo ARCUS HBV Program has Shown Exciting Efficacy Data During In Vivo POC Gene Excision for Studies Demonstrating Ability to Reduce Both HBV S-Antigen and HBV cccDNA Chronic Hep B > 200 150 (% of baseline) Data from in vivo models1 treated with ARCUS-HBV HBsAg PBS 100 nuclease via LNP showed pot
Slide 24: ARCUS to Restore Dystrophin Expression for Duchenne Muscular Dystrophy
In Vivo ARCUS to Restore Dystrophin Expression for Duchenne Muscular Dystrophy Gene Excision for DMD > Restore dystrophin expression Deleting exons 45-55 using a pair of ARCUS nucleases intended to remove a mutation hotspot responsible for >50% of DMD ARCUS 1 ARCUS 2 GOAL Exon 44 Exons 45–55 Exon 56
Slide 25: 2023 Priorities
PRECISION BIOSCIENCES 2023 Priorities
Slide 26: 2023 Priorities and Milestones
2023 Priorities and Milestones 〉Complete Phase 1b for azer-cel to support decision point for Phase 2 〉Complete Phase 1 dosing trial for PBCAR19B 〉Host CAR T update in May 2023 〉Nominate the final drug candidate for HBV in vivo program 〉Advance the first ARCUS in vivo nuclease to CTA through a partne
Slide 27: Building the leading therapeutic gene editing company
Technology: ARCUS, a premier genome editing platform Building the leading People: Fortified Senior Leadership therapeutic gene editing Team with 15+ years perfecting company focused on high ARCUS protein engineering unmet needs in genetic diseases Focus & Discipline: Fiscal resources support 2+ year
Slide 28: Senior Leadership Team
Precision BioSciences: Senior Leadership Team Michael Amoroso Jeff Smith, Alex Kelly Cindy Atwell Since Sep 2021 Ph.D. Since Oct 2020 Since Apr 2019 President & Since Mar 2006 Chief Financial Officer Chief Business Officer Chief Executive Officer Chief Research Officer, Co-Founder Alan List, M.D. Ju
Slide 29: Board of Directors
Precision BioSciences: Board of Directors Kevin Buehler Michael Amoroso Melinda Brown Stanley Frankel, M.D. Since Jul 2019 Since Oct 2021 Since May 2022 Since Apr 2021 Board Chair Audit Chair Science & Technology Chair Geno Germano Shari Lisa Piré, J.D. Sam Wadsworth, Ph.D. Since Mar 2020 Since Nov
Slide 30: Appendix
PRECISION BIOSCIENCES Appendix
Slide 31: ARCUS for Chronic Hepatitis B Virus Targeting cccDNA
In Vivo ARCUS for Chronic Hepatitis B Virus (cHBV) Targeting cccDNA Gene Excision for Chronic Hep B ARCUS-mediated inactivation of cccDNA and integrated HBV could result in a functional cure ARCUS HBV Infected Hepatocyte LNP Chronic HBV is one of greatest racial health disparities in the U.S. AAA >
Slide 32: In Vivo Gene Editing Partnership with Prevail
In Vivo Precision’s In Vivo Gene Editing Partnership with Prevail Gene Excision for DMD Research collaboration and license agreement aimed at treating + challenging genetic diseases Pre-IND R&D IND to Commercial 3 Initial collaboration for three targets, including Duchenne muscular dystrophy and two
Slide 33: Duchenne Muscular Dystrophy Currently Lacks a Curative Treatment
In Vivo Duchenne Muscular Dystrophy Currently Lacks a Curative Treatment Gene Excision for DMD Mutation on the X chromosome interferes On average, with dystrophin protein production, which children lose is needed to form and maintain healthy muscle their ability to walk by Affects approximately age
Slide 34: In Vivo Gene Insertion Collaboration with Novartis for Hemoglobinopathies
In Vivo In Vivo Gene Insertion Collaboration with Novartis for Hemoglobinopathies Gene Insertion for Hemoglobinopathies Builds on the unique gene insertion capabilities + of ARCUS® and further validates ARCUS as a Develop single Preclinical to premium genome editing platform ARCUS nuclease commercia
Slide 35: Hemoglobinopathies are a Major World Health Problem
In Vivo Hemoglobinopathies are a Major World Health Problem Gene Insertion for Hemoglobinopathies Sickle Cell Disease (SCD) Beta Thalassemia Affects the structure/function of hemoglobin, One of the most common genetic diseases caused by a disruption reducing the ability of red blood cells to transpo
Slide 36: OTC is a Severe, Ultra Rare Genetic Condition
In Vivo OTC is a Severe, Ultra Rare Genetic Condition With Extremely High Unmet Gene insertion Medical Needs Across Phenotypes for OTC Ornithine Transcarbamylase (OTC) Deficiency ~ 4,200 People with OTC in the US2 • OTC deficiency is the most • OTC deficiency is also associated Neonatal-onset Late-o
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