Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — Investor Day (September 2021)
76 slides from Precision Biosciences's investor day presentation, Sep 9, 2021
Slide 1: Virtual Gene Editing R&D Event
Virtual Gene Editing R&D Event September 9, 2021
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation (together with any other statements or information that we may make in connection herewith) may contain forward-looking statements. All statements other than statements of present and historical facts contained in this prospectus, including without limita
Slide 3: Precision & Versatility; 3 INDs/CTAs in 3 Years
3 1 PRECISION 2 NEW Collaboration & VERSATILITY 3 INDs/CTAs focused on gene insertion & accelerating clinical ARCUS has the potential to address in 3 YEARS validation of ARCUS a broader spectrum of genetic diseases 3
Slide 4: Agenda
Intro to ARCUS for in vivo Gene Editing Gene Editing Pipeline AGENDA: & Strategy The Next Frontier 4
Slide 5: Intro to ARCUS for in vivo Gene Editing
INTRO TO ARCUS FOR in vivo GENE EDITING 5
Slide 6: Chlamydomonas reinhardtii
Chlamydomonas reinhardtii 6
Slide 7: ARCUS is derived from I-CreI, a homing endonuclease from algae
I-CreI is 1 expressed 23S rRNA gene I-CreI 5’-CAAACTGTCGTGAGACGTTTTG-3’ 2 recognizes its target in the ARCUS is derived 3’-GTTTGACAGCACTCTGCAAAAC-5’ 23S rRNA gene from I-CreI, a homing endonuclease from algae 3 produces I-CreI cuts and 3’ 5’-CAAACTGTCGTGA GACGTTTTG-3’ overhangs 3’-GTTTGACAG CACTCTGC
Slide 8: ARCUS: Custom Engineered from I-CreI
ARCUS: Custom Engineered from I-CreI I-CreI can be redesigned to edit new DNA sequences I-CreI ARCUS (Homodimer) (Single Chain) monomer 1 monomer 2 Protein Engineering 5’-CAAACTGTCGTGA GACGTTTTG-3’ 5’-TGAACTGAATCGA TTGCACGAT-3’ 3’-GTTTGACAG CACTCTGCAAAAC-5’ 3’-ACTTGACTTA AGCTAACGTGCTA-5’ “Left” half
Slide 9: Creating and Optimizing ARCUS Nucleases
Creating and Optimizing ARCUS Nucleases The DNA-binding surface of I-CreI can be extensively re-engineered to produce each new ARCUS nuclease Controls Controls Controls Efficiency Specificity Affinity 9
Slide 10: Creating and Optimizing ARCUS Nucleases
Site Selection I-CreI In silico Directed Design Evolution Protein Engineering 1st Generation ARCUS Creating and Optimizing Off-Target ARCUS Nucleases Analysis Lead Generation Next Generation Repeat Optimization Desired Therapeutic Index Achieved? ARCUS Cycle 2-3X NO YES Lead Optimization Specificity
Slide 11: Precision and Versatility
PRECISION • Safety VERSATILITY • Specificity • ARCUS is Easy to Deliver • ARCUS Performs Complex Edits (Gene Insertion & Gene Repair) 11
Slide 12: Safety and Specificity
Safety and Specificity ARCUS is inactive until it binds to its target DNA site This allows ARCUS to be expressed for extended periods of time without accumulating off-target gene edits. Inactive Form of ARCUS closed configuration buries the active site inside the protein 12
Slide 13: Safety and Specificity
Safety and Specificity ARCUS is inactive until it binds to its target DNA site This allows ARCUS to be expressed for extended periods of time without accumulating off-target gene edits. Active Form of ARCUS open configuration allows the active site to access DNA active site 13
Slide 14: Safety and Specificity: Off-target editing
Safety and Specificity Off-target editing: Detection is the challenge • The number of off-target sites identified is a function of how well they can be detected. • Off-target sites are very difficult to detect for most editing platforms. • If you can detect better, you can engineer a better editing
Slide 15: Oligo Capture: genome-wide off-target assay
Safety and Specificity 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’-CGCTGCTAGCTGATGCGCTAGTAGCT GCTAGTCGCTAGTCGGC-
Slide 16: Oligo Capture more sensitive than CRISPR assays
Safety and Specificity Oligo Capture has been shown to be far more sensitive than assays developed for CRISPR Wang, et al. (2018 Nat. Biotechnol. 36(8):717-725) used both Oligo Capture and GUIDE-seq (the “gold standard” method for CRISPR) to locate sites of off-target editing in non-human primates t
Slide 17: ARCUS Is Easy to Deliver
ARCUS Is Easy to Deliver The small size of ARCUS makes it compatible with single AAV delivery AAV Genome Capacity (~ 4700 bp) ARCUS P ARCUS nuclease ITR NLS pegRNA P ARCUS nuclease 1 P ARCUS nuclease 2 U Promoter polyA UGI P Promoter gRNA CRISPR/Cas U P SaCas9 U P AsCas12a/Cpf1 U P SpCas9 Prime Edit
Slide 18: DNA Cuts Can Be Repaired by Either NHEJ or HDR
DNA Cuts Can Be Repaired by Either NHEJ or HDR. Complex gene insertion or gene repair edits require HDR. NHEJ HDR Non-Homologous End-Joining Homology-Directed Repair Gene Knockout Gene Insertion or Repair 18
Slide 19: ARCUS Performs Complex Edits
ARCUS Performs Complex Edits 20.7% ARCUS Promotes HDR Indel Cuts made by ARCUS have 3’ overhangs and are repaired primarily via HDR This enables complex edits like gene insertion 49.4% Gene Insertion Precision BioSciences’ CAR T Engineering Process 19
Slide 20: Blunt Cuts are Repaired Primarily via NHEJ
Blunt Cuts are Repaired Primarily via NHEJ 58.0% 3’ overhangs are Indel necessary for efficient HDR Blunt cut 6.6% Gene Insertion 20
Slide 21: ARCUS Performs Complex Edits: HDR:NHEJ Ratio
ARCUS Performs Complex Edits 2.5 HDR:NHEJ Ratio 2 The 3’ overhangs created by ARCUS very significantly Ratio of HDR:NHEJ 1.5 shift the ratio of HDR:NHEJ ~20X difference to favor HDR in HDR:NHEJ 1 We believe ARCUS is the 0.5 ideal tool for gene insertion and repair. 0 3’ overhang Blunt End 21
Slide 22: Intellectual Property
Intellectual Property • Precision BioSciences controls over 80 issued patents related to ARCUS and its applications • ARCUS platform and nucleases are unencumbered by third-party IP Applications and Issued Patents by Year Intellectual Property Distribution 500 90 450 80 Total Applications Filed 400
Slide 23: Two In-House Delivery Platforms: LNP
Two In-House Delivery Platforms: LNP & AAV Efficient delivery to liver LNP ARCUS mRNA Short half-life No neutralizing antibodies Lipid Nanoparticle: Potential to repeat-dose AAA Good translation from NHP * *Precision has non-exclusive rights to evaluate Acuitas Therapeutics, Inc.’s LNP technology fo
Slide 24: Two In-House Delivery Platforms: AAV
Two In-House Delivery Platforms: LNP & AAV Delivery to other tissues AAV Adeno-Associated Virus: ARCUS DNA Deliver HDR donor for knock-in Established manufacturing Established regulatory pathway Promoters localize expression 24
Slide 25: LNP Biodistribution and Pharmacokinetics
LNP Biodistribution and Pharmacokinetics LNP Delivers ARCUS Efficiently to NHP Liver and Dissipates in Days Lipid Nanoparticle (LNP) 2 mg/kg IV 1 hour 3 hour 6 hour 24 hour 48 hour 7 day ARCUS mRNA DAPI 25
Slide 26: AAV Biodistribution and Pharmacokinetics
AAV Biodistribution and Pharmacokinetics AAV Delivers ARCUS Efficiently to NHP Liver 17 day 129 day and Persists Longer than LNP Adeno-Associated Virus (AAV) 3e13 vg/kg AAV8 IV Wang, et al. (2018) Nat. Biotechnol. 36(8):717-725 ARCUS mRNA 26
Slide 27: MCAT: Manufacturing Center for Advanced Therapeutics
MCAT: Manufacturing Center for Advanced Therapeutics 17,300 sq. ft. facility in Durham, NC Fully cGMP compliant CAR T, mRNA, and AAV platforms Currently producing clinical trial material for CAR T programs 27
Slide 28: Directions for Q&A
To ask a question, please use dial-in conference call numbers Directions • (866) 970-2058 DOMESTIC • (873) 415-0216 INTERNATIONAL for Q&A: The conference ID number for the call is 6376435 When asking a question, please mute your webcast video. 28
Slide 29: ARCUS Q&A
ARCUS Q&A 29
Slide 30: Gene Editing Pipeline & Strategy
GENE EDITING PIPELINE & STRATEGY 30
Slide 31: Gene Editing Pipeline
Gene Editing Pipeline Candidate IND- Expected Program Indication Tissue Target Delivery Research Partner Selection Enabling IND/CTA PBGENE- Familial Liver PCSK9 AAV 2022 PCSK9* Hypercholesterolemia PBGENE- Primary Liver HAO1 LNP 2023 PH1 Hyperoxaluria Type 1 PBGENE- Chronic Hepatitis B Liver HBV LNP
Slide 32: Gene Editing Pipeline Validation & Expansion Creates Value
Gene Editing Pipeline 22 Validation & Expansion Pipeline Expansion Creates Value Complex Edits & 1 Multiple Tissues Clinical Validation Knock-out Genes in Liver PBGENE-DMD PBGENE-LLY2* AAV PBGENE-PCSK9 PBGENE-LLY3* LNP PBGENE-PH1 PBGENE-HBV IND/CTA 2022 2023 2024 Submission *Precision has not disclo
Slide 33: In Vivo Gene Editing Partnering Strategy
In Vivo Gene Editing Partnering Strategy Precision Biosciences Programs Partnering Strategy to Build Precision Scale Speed Leverage extensive NHP data to achieve clinical validation of liver knock-out programs Economics Where Precision Biosciences can be 1st gene editing program into clinic Capabili
Slide 34: Transformative Gene Editing Partnership for Precision
Transformative Gene Editing Partnership for Precision Research collaboration and license agreement aimed at treating challenging genetic diseases 3+ Initial collaboration for 3 programs, including DMD Precision - Pre-IND R&D; Lilly - IND to commercial Upfront payment of $135 million including 3 Lill
Slide 35: iECURE Collaboration Provides Potential Rapid Path to Clinical Validation
iECURE Collaboration Provides Potential Rapid Path to Clinical Validation Collaboration leverages extensive ARCUS knock-out data in NHPs, rapidly advances first ARCUS nuclease into the clinic, and validates ARCUS for gene insertion Rapid path for PBGENE-PCSK9 through Partnered with the Gene clinical
Slide 36: PCSK9 Familial Hypercholesterolemia (FH)
DEVELOPMENT PROGRAMS: PCSK9 FAMILIAL HYPERCHOLESTEROLEMIA (FH) 36
Slide 37: Familial Hypercholesterolemia
Familial Hypercholesterolemia Hypercholesterolemia PCSK9 Knockout LDL LDL PCSK9 LDL-R LDL-R PCSK9 secreted Hepatocyte LDL-R recycled LDL-R degraded Endocytosis Endocytosis 37
Slide 38: PBGENE-PCSK9: Stable Knockout of PCSK9 Observed in NHPs
PBGENE-PCSK9: Stable Knockout of PCSK9 Observed in NHPs A single dose of AAV8-ARCUS reduced serum PCSK9 levels by up to 82% in non-human primates 0 2e12 vg/kg PCSK9 (% reduction from baseline) -20 6e12 vg/kg 3e13 vg/kg 30% -40 Reduction 49% -60 Reduction 60% Wang, et al. (2021) Mol. Ther. 29(6):2019
Slide 39: PBGENE-PCSK9: Stable Reduction in LDL-c Observed in NHPs
PBGENE-PCSK9: Stable Reduction in LDL-c Observed in NHPs A single dose of AAV8-ARCUS reduced LDL-c levels by up to 62% in non-human primates 0 2e12 vg/kg LDL-C (% reduction from baseline) -20 6e12 vg/kg 3e13 vg/kg -40 39% 42% Reduction Reduction 52% -60 Reduction 62% Wang, et al. (2021) Mol. Ther. 2
Slide 40: PBGENE-PCSK9 Vector
Research Vector • Wang, et al. (2018) Nat. Biotechnol. 36(8):717-725 • Wang, et al. (2021) Mol. Ther. 29(6):2019-2029 AAV8 PBGENE-PCSK9 Vector TBG M2PCSK9 WPRE BGH AAV was selected as the promoter ARCUS enhancer poly-A delivery technology for PBGENE-PCSK9 • The underlying genetic causes of FH Antici
Slide 41: HAO1 Primary Hyperoxaluria Type 1
DEVELOPMENT PROGRAMS: HAO1 PRIMARY HYPEROXALURIA TYPE 1 41
Slide 42: PBGENE-PH1
PBGENE-PH1 Primary Hyperoxaluria Type 1 is a potentially fatal genetic disease caused by a gene mutation that leads to the accumulation of calcium oxalate crystals in the kidneys. Glycolaldehyde Glycolate Glyoxylate AGXT LDH Oxalate Calcium Oxalate Glycine 42
Slide 43: PBGENE-PH1
PBGENE-PH1 Primary Hyperoxaluria Type 1 is a potentially fatal genetic disease caused by a gene mutation that leads to the accumulation of calcium oxalate crystals in the kidneys. Suppressing expression of the HAO1 gene is a proven method for treating the disease by reducing the formation of calcium
Slide 44: PBGENE-PH1 Formulation
Research Vector AAV8 TBG M1HAO1 WPRE BGH promoter ARCUS enhancer poly-A PBGENE-PH1 Formulation LNP was selected as the Anticipated Clinical Formulation delivery technology for PBGENE-PH1 • Generation 6 ARCUS nuclease for HAO1 • Lipid nanoparticle formulation with optimized mRNA sequence M6HAO1 AAA 4
Slide 45: ARCUS Reduced HAO1 mRNA levels
ARCUS Reduced HAO1 mRNA levels 1.2 1 Normalized HAO1 mRNA (n=3) PBGENE-PH1 0.8 Proof of Concept in Non-Human Primates 0.6 0.4 ARCUS treatment decreased HAO1 mRNA by 98% in NHPs 0.2 98.0% reduction AAV Dose 3e13 vg/kg (n=3) 0 CONTROL ARCUS (M1HAO1) 45
Slide 46: ARCUS Reduced GO Protein Levels
ARCUS Reduced GO Protein Levels 1.2 CONTROL ARCUS (M1HAO1) 1 NHP-1 NHP-2 NHP-1 NHP-2 NHP-3 Normalized GO protein (n=3) PBGENE-PH1 vinculin 0.8 Proof of Concept 0.6 97.9% 0.4 in Non-Human Primates 0.2 reduction ARCUS treatment decreased GO 0 CONTROL ARCUS GO protein1 by 97.9% in NHPs (M1HAO1) AAV Dos
Slide 47: PBGENE-PH1 Proof of Concept in Non-Human Primates
PBGENE-PH1 Proof of Concept in Non-Human Primates Serum glycolate siRNA ARCUS treatment significantly increased serum glycolate Published serum glycolate levels in NHPs treated with levels in non-human primates an siRNA against HAO1 (Alnylam, PCT/US2015/054881) 100 ARCUS (M1HAO1) single dose 80 Seru
Slide 48: PBGENE-PH1: Efficiency of HAO1 gene editing
Efficiency of HAO1 gene editing 100 90 80 PBGENE-PH1: 70 Development Candidate % On-target Editing 60 M6HAO1 (candidate) 50 M1HAO1 (research) 40 On-Target Editing: The optimized ARCUS 30 candidate (M6HAO1) had comparable on-target editing efficiency to the research 20 nuclease in transfected cells i
Slide 49: PBGENE-PH1: Off-Target Editing
PBGENE-PH1: site was identified in human cells Development Candidate Off-Target Editing: One potential off-target transfected with 100ng of M6HAO1 mRNA. Indel % above background 0 1 0.1 0.2 0.3 0.4 0.5 0.6 0.7 0.8 0.9 chr1_175654632_175654654 chr1_182562795_182562817 chr1_194256892_194256914 chr10_9
Slide 50: HBV Chronic Hepatitis B
DEVELOPMENT PROGRAMS: HBV CHRONIC HEPATITIS B 50
Slide 51: PBGENE-HBV Therapeutic Strategy
ARCUS ARCUS HBV Infected Hepatocyte LNP AAA PBGENE-HBV Therapeutic Strategy inactivate cccDNA ARCUS-mediated inactivation of cccDNA and integrated HBV inactivate could result in a functional cure integrated HBV Durable Antigen Loss Functional Cure 51
Slide 52: Antiviral Activity in HBV-Infected Human Hepatocytes
Antiviral Activity in HBV-Infected Human Hepatocytes cccDNA Indels HBsAg ARCUS treatment resulted in significant Indel mutations were detected in the ARCUS treatment resulted in significant reductions in total HBV cccDNA remaining HBV genomes following reductions in secreted S-antigen ARCUS treatmen
Slide 53: Challenge: There is No in vivo Model of Human HBV Infection
Challenge: There is No in vivo Model of Human HBV Infection 1. HBV Genome 2. 1. AAV HBVsAg 2. ARCUS LNP We developed a novel in vivo model for HBV editing 2. 1. HBV genome sequences are delivered on HBsAg an AAV vector. 2. HBV sequences are then deleted by ARCUS ARCUS LNP delivered by LNP. sAg RNA 2
Slide 54: Mouse Episomal Model Results
Mouse Episomal Model Results LNP-ARCUS efficiently edited HBV sequences in an immunodeficient mouse model AAV Copy Number Indels HBsAg ARCUS treatment resulted in significant ARCUS treatment introduced a high ARCUS treatment resulted in a reductions in total AAV genome copies frequency of indel muta
Slide 55: NHP Episomal Model Results
NHP Episomal Model Results LNP-ARCUS efficiently edited HBV sequences in a NHP model AAV Copy Number Indels ARCUS treatment resulted in significant ARCUS treatment introduced a high frequency of reductions in total AAV genome copies indel mutations into the remaining AAV genomes 50 60 50 40 Copies/D
Slide 56: DMD Duchenne Muscular Dystrophy
DEVELOPMENT PROGRAMS: DMD Duchenne Muscular Dystrophy 56
Slide 57: ARCUS for Therapeutic Treatment of DMD
ARCUS for Therapeutic Treatment of DMD ARCUS 1 ARCUS 2 Goal: Restore dystrophin Exons 45–55 expression by deleting exons Exon 44 Exon 56 Exon 57 45-55 using a pair of ARCUS nucleases intended to remove a mutation hotspot Exons 45–55 deletion responsible for >50% Exon 44 Exon 56 Exon 57 of DMD genome
Slide 58: Dystrophin Gene Correction Observed in DMD Patient Myoblasts
Dystrophin Gene Correction Observed in DMD Patient Myoblasts 40 % Editing (“perfect ligation”) 30 20 Corrected DNA Exons 45-55 deleted 10 0 mock ARCUS mRNA 58
Slide 59: Dystrophin Gene Correction Observed in DMD Patient Myoblasts
Dystrophin Gene Correction Observed in DMD Patient Myoblasts 10 8 (normalized to ANKRD27) Spliced DMD mRNA 6 Corrected mRNA 4 Exon 44 spliced to Exon 56 2 0 mock ARCUS mRNA 59
Slide 60: Dystrophin Gene Correction Observed in DMD Patient Myoblasts
Dystrophin Gene Correction Observed in DMD Patient Myoblasts kDa 440- 280- Dystrophin Corrected Protein 200- Dystrophin protein expressed er k oc dd m la ARCUS mRNA 60
Slide 61: The Next Frontier
The Next Frontier 61
Slide 62: Mitochondrial Diseases
Mitochondrial Diseases • Mitochondria are the powerhouse of the cell Alpers Disease: Progressive Infantile Poliodystrophy Barth Syndrome: Lethal Infantile Cardiomyopathy • Pathogenic mutations in Complex (I-V) Deficiency mitochondrial genome reduce the Co-Enzyme Q10 Deficiency ability to generate en
Slide 63: Mitochondrial Genome Editing
Mitochondrial Genome Editing ARCUS has selectively eliminated mutant mitochondrial genomes that cause disease in preclinical studies AAV MTS ARCUS BGH promoter Mito ARCUS poly-A Targeting WT mtDNA TS Mitochondrial M matrix Mutant mtDNA Cytoplasm 63
Slide 64: Mitochondrial Genome Editing
Mitochondrial Genome Editing ARCUS has selectively eliminated mutant mitochondrial genomes that cause disease in preclinical studies AAV MTS ARCUS BGH promoter Mito ARCUS poly-A Targeting WT mtDNA TS Mitochondrial M Mutant matrix DNA Deleted Cytoplasm 64
Slide 65: Mitochondrial Genome Editing
Mitochondrial Genome Editing ARCUS fused to a Mitochondrial Targeting Sequence (MTS) localized to mitochondria in vitro Zekonyte, et al (2021) Nat Commun 12(1):3210 65
Slide 66: Mitochondrial Genome Editing
Mitochondrial Genome Editing ARCUS restored markers of mitochondrial function in a cell model of mitochondrial disease ARCUS Deleted Mutant Genomes ARCUS Improved Respiration ARCUS Increased ATP Production Hybrid cells were converted to >99% WT Elimination of defective genomes improved Restoration o
Slide 67: Mitochondrial Genome Editing
Mitochondrial Genome Editing AAV-delivered ARCUS eliminated mutant mitochondrial genomes in a mouse model Reduction in mutant genomes following systemic AAV9-ARCUS delivery (4e9 vg/kg) 25 % Change mutant mtDNA 0 (Normalized to Brain) -25 Control ARCUS -50 -75 -100 Heart Tibialis Quad Calf Kidney Liv
Slide 68: Gene Insertion into the PCSK9 locus in Newborn and Infant NHPs
Gene Insertion into In collaboration with the the PCSK9 locus University of Pennsylvania in Newborn and Gene Therapy Program Infant NHPs 68
Slide 69: ARCUS-mediated hFIX Gene Targeting in Newborn or Infant NHPs
ARCUS-mediated hFIX Gene Targeting in Newborn or Infant NHPs IV (Nuclease + hFIX HDR donor) Newborn or birth infant rhesus 1st Liver biopsy 2nd Liver biopsy d0 d14 d28 d43 d56 d84 (newborn study) 1y d98 (infant study) Newborn study: 2 days Infant study: 3 months BGH Promoter ARCUS2 WPRE Exon 7 ITR I
Slide 70: CRISPR/Cas9-mediated hFIX Gene Targeting in Newborn or Infant NHPs
CRISPR/Cas9-mediated hFIX Gene Targeting in Newborn or Infant NHPs IV (Nuclease + hFIX HDR donor) Newborn or birth infant rhesus 1st Liver biopsy 2nd Liver biopsy d0 d14 d28 d43 d56 d84 (newborn study) 1y d96 (infant study) Newborn study: 2 days Infant study: 3 months BGH or GFP BGH Promoter SaCas9
Slide 71: Nuclease-mediated hFIX Gene Targeting in Newborn NHPs
Nuclease-mediated hFIX Gene Targeting in Newborn NHPs Normal level 71
Slide 72: Nuclease-mediated hFIX Gene Targeting in Infant NHPs
Nuclease-mediated hFIX Gene Targeting in Infant NHPs Normal level 72
Slide 73: Nuclease-mediated Gene Targeting in Newborn and Infant NHPs
Nuclease-mediated Gene Targeting in Newborn and Infant NHPs hFIXco ISH – Digitalized images for quantification of transduction % Newborn Infant ARCUS day 84 ARCUS day 366 ARCUS day 98 ARCUS SaCas9 day 151 SaCas9 day 96 SaCas9 GFP day 84 untreated day 84 Control 73
Slide 74: Nuclease-mediated Gene Targeting in Newborn and Infant NHPs
Nuclease-mediated Gene Targeting in Newborn and Infant NHPs % Gene Addition by dual ISH 25 20.1% 20 WPRE (ARCUS, SaCas9, or GFP Vector Inserted) 15 % ISH+ Cells hFIX 12.1% 11.6% (hFIX Vector Inserted) 10 WPRE & hFIX (Both Vectors Inserted) 5 1.6% 0.8% 0.1% 0 Day 84 Day 366 Day 98 Day 151 Day 96 Day
Slide 75: Conclusions
Conclusions • Transgenes were targeted efficiently to the PCSK9 locus using ARCUS in newborn and infant NHPs • Gene addition appeared to be stable over time • This may represent a “universal” approach to treating rare genetic diseases caused by loss-of-function mutations in liver 75
Slide 76: Precision & Versatility; 3 INDs/CTAs in 3 Years
3 1 PRECISION 2 NEW Collaboration & VERSATILITY 3 INDs/CTAs focused on gene insertion & accelerating clinical ARCUS has the potential to address in 3 YEARS validation of ARCUS a broader spectrum of genetic diseases
Precision BioSciences — Investor Day (September 2021) on 6ix