Editas Medicine, Inc. · NASDAQ:EDIT
Editas Medicine — Investor Presentation (May 2019)
30 slides from Editas Medicine's investor presentation, May 9, 2019
Slide 1: Corporate Presentation
Corporate Presentation May 9, 2019 PIONEERING THE POSSIBLE editasmedicine.com © 2019 Editas Medicine 1
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements within the meaning of the “safe harbor” provisions of The Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, contained in this presentation, including statements
Slide 3: Transforming Patients' Lives
Transforming Patients’ Lives OUR MISSION is to translate the promise of genome editing into a broad class of transformative genomic medicines to benefit the greatest number of patients © 2019 Editas Medicine 3
Slide 4: Pioneering the Possible
Pioneering the Possible Clinical-stage company Leader in CRISPR Strong progress working to transform in vivo and engineered against EM22 patients’ lives cell medicines goals • First in vivo CRISPR • Proven AAV delivery • EDIT-101 IND accepted by clinical trial in history capability for CRISPR FDA up
Slide 5: EM22: Our 2022 Vision for Editas Medicine
EM22: Our 2022 Vision for Editas Medicine ≥ Medicines In ≥ Medicines In Best-in-Class: 3 Early-Stage Clinical Trials 2 or Ready for Late-Stage Clinical Trials # 1 Medicines Platform Culture Build on Our Current Success Establish New Areas & Leverage Our Platform At least one program from our At leas
Slide 6: Developing Best-in-Class CRISPR Medicines
Developing Best-in-Class CRISPR Medicines EDITING INSIDE THE BODY EDITING OUTSIDE THE BODY IN VIVO CRISPR MEDICINES ENGINEERED CELL MEDICINES OCULAR DISEASES CANCER Leber congenital amaurosis 10* T cells for solid & liquid tumors** Usher syndrome 2A Allogeneic cell medicines Retinitis pigmentosa Ocu
Slide 7: Durable Medicines for Serious Eye Diseases
Durable Medicines for Serious Eye Diseases Retina LEBER CONGENITAL AMAUROSIS Hundreds of thousands USHER SYNDROME of patients RETINITIS PIGMENTOSA Cone-rod dystrophy Age-related macular degeneration Targeted local injection Stargardt disease with proven AAV vectors Promising clinical and regulatory
Slide 8: Targeting Leading Genetic Form of Blindness
Targeting Leading Genetic Form of Blindness Remove genetic mutation to restore CEP290 protein and rebuild photoreceptors in Leber congenital amaurosis 10 First patient dosing expected in 2H19 2,000 - 5,000 and Degeneration patients of photoreceptors leading to in US and blindness in Europe 50/50 in
Slide 9: EDIT-101 Aims to Rescue Vision in LCA10
EDIT-101 Aims to Rescue Vision in LCA10 LCA10 Photoreceptor EDIT-101 Rescued Photoreceptor Outer Segment Outer Segment Outer segment Editing removes Outer segment degenerates due to disease-causing regenerates with CEP290 deficiency mutation CEP290 protein © 2019 Editas Medicine 9
Slide 10: EDIT-101 Aims to Rescue Vision in LCA10
EDIT-101 Aims to Rescue Vision in LCA10 Chromosome 12 position q21.32 Mutated CEP290 Gene EDIT-101 PAM Mutation Genome Editing Deletion Cut and Remove Repaired CEP290 Gene Transcription and normal mRNA splicing Protein translation Restored CEP290 Protein © 2019 Editas Medicine 10
Slide 11: EDIT-101 Demonstrates Rapid Editing
EDIT-101 Demonstrates Rapid Editing CEP290 GENE EDITING MOUSE FULL-THICKNESS RETINA Rapid editing in transgenic mice 100 carrying human gene mutation Editing (%) - logarithmic scale Rapid editing also observed in non-human primates 10 Editing in both species well above predicted therapeutic threshol
Slide 12: EDIT-101 is Well Tolerated in Non-Human Primates
EDIT-101 is Well Tolerated in Non-Human Primates 4 4 AQUEOUS FLARE AQUEOUS CELLS LEGEND 3 3 Clinical Score Clinical Score EDIT-101 2 2 Placebo 1 1 Animal 1 Animal 2 0 0 3 8 15 29 46 53 65 92 9 3 8 15 29 46 53 65 92 9 Animal 3 -1 -1 Day post dosing Day post dosing Day Day 4 4 VITREOUS HAZE VITREOUS C
Slide 13: Setting the Stage for Interventional Trials
Setting the Stage for Interventional Trials LCA10 NATURAL HISTORY STUDY PATIENTS ~40 patients, aged 3 and above Characterize patients, assessments, OBJECTIVES rate of change and validate endpoints SITES 7 sites across US and Europe 6 visits over 1 year; preliminary FOLLOW-UP data presented in April
Slide 14: EDIT-101 Advancing to Clinical Trials
EDIT-101 Advancing to Clinical Trials EDIT-101 PHASE 1/2 TRIAL Open-label, dose escalation study DESIGN to evaluate safety, tolerability, and efficacy PATIENTS ~18 patients with IVS26 mutation* Patient’s own baseline value COMPARATOR for each efficacy measure Core measurements FOLLOW-UP every 3 mont
Slide 15: Pursuing Usher Syndrome 2A Medicine
Pursuing Usher Syndrome 2A Medicine Rescue vision by restoring USH2A protein leveraging same proprietary enzyme, vector, and promoter Editing approach as EDIT-101 validated in collaboration with Drs. Eric Pierce and Qin Liu 4,000 Progressive patients with vision loss leading target mutation to blind
Slide 16: Editing USH2A Increases Cilia Length in vivo
Editing USH2A Increases Cilia Length in vivo Demonstrates that editing approach results in desired phenotype Pendse et al., CRISPR/Cas Based Evaluation of the Therapeutic Potential for USH2A Associated Diseases, American Society of Gene & Cell Therapy 22nd Annual Meeting © 2019 Editas Medicine 16
Slide 17: Developing Transformative Engineered Cell Medicines
Developing Transformative Engineered Cell Medicines Hematopoietic stem cells could yield multiple medicines for blood diseases T cells and NK cells are therapeutic platform for cancer, autoimmune, and infectious diseases Editas editing enables both autologous and allogeneic cell medicines NK: natura
Slide 18: Towards Durable Sickle Cell Disease Medicine
Towards Durable Sickle Cell Disease Medicine Edit hematopoietic stem cells to increase fetal hemoglobin and alleviate morbidity and mortality of disease Distinct editing strategy designed to deliver Over best-in-class Sickle cell 100,000 disease and medicines beta-thalassemia hospitalizations causin
Slide 19: Harnessing Genetics to Treat Sickle Cell Disease
Harnessing Genetics to Treat Sickle Cell Disease Three Critical Product Candidate Criteria 1 SUCCESSFUL 2 3 EDITING MAINTENANCE DURABLE of long-term of normal HSC hematopoietic predicted therapeutic function stem cells induction of fetal hemoglobin Editing strategy aligns with human genetics and is
Slide 20: Editas Approach Maintains HSC Function
Editas Approach Maintains HSC Function GLOBIN LOCUS EDITING BCL11Ae EDITING EDITAS APPROACH COMPETING APPROACH SIMILAR LEVELS OF SIMILAR LEVELS REDUCED INCREASED ERYTHROID OUTPUT OF CELL DEATH ERYTHROID OUTPUT CELL DEATH 120 6 120 *** 6 *** 100 100 % Control (Erythroid) % Control (Erythroid) % Caspa
Slide 21: Editas Approach Induces Robust Fetal Hemoglobin
Editas Approach Induces Robust Fetal Hemoglobin HbF INDUCTION ex vivo HbF INDUCTION in vivo CD34+ CELLS RED BLOOD CELL PRECURSORS 50 50 40 40 HbF / (HbF + HbA) (%) HbF / (HbF + HbA) (%) 30 30 20 20 10 10 0 0 Control HBG1/2 Control HBG1/2 Potential to safely and effectively induce fetal hemoglobin in
Slide 22: Editas Approach Achieves Product Candidate Criteria
Editas Approach Achieves Product Candidate Criteria 1 SUCCESSFUL 2 3 EDITING MAINTENANCE DURABLE of long-term of normal HSC hematopoietic predicted therapeutic function stem cells induction of fetal hemoglobin BCL11Ae ✓ ✗ GLOBIN LOCUS ✓ ✓ ✓ © 2019 Editas Medicine 22
Slide 23: Engineered T Cells for Cancer
Engineered T Cells for Cancer Expand range of cancers that can be treated with Editas Medicine engineered CAR T and CRISPR edited TCR cell medicines product candidates advancing in both solid and Initial collaboration liquid tumors Achieved milestones highly efficient editing of multiple achieved ge
Slide 24: Powerful Engine for Genomic Medicines
Powerful Engine for Genomic Medicines Broadest Access to Genomic Targets Proprietary portfolio of Cas9 and Cpf1 enzymes may directly edit ~95% of the human genome Widest Range of Tissues and Cells Ability to use AAVs, RNPs, and LNPs to address diseases throughout the body Diverse Spectrum of Therape
Slide 25: Strong Foundation for Long-Term Leadership
Strong Foundation for Long-Term Leadership PARTNERS PATENTS OCULAR ENGINEERED T CELL BROADEST & DEEPEST MEDICINES MEDICINES FOR CANCER PORTFOLIO OF CRISPR IP Option to license up to CAR T and TCR cell medicines to Exclusive access to foundational 5 ocular programs treat cancer Cas9 and Cpf1 patent e
Slide 26: Appendix
Community Resilience Ingenuity Appendix Science Passion Revolution www.editasmedicine.com © 2019 Editas Medicine 26
Slide 27: Platform Enables Broad Product Pipeline
Platform Enables Broad Product Pipeline ~10x Broad Range of Sites SpCas9 SaCas9 SpCas9 SaCas9 Cpf1 Cpf1 Editas Variants Variants Variants Platform Wide Delivery Options Viral Vector Lipid Nanoparticle Electroporation Disrupt Remove Replace Insert Diverse Spectrum of Edits © 2019 Editas Medicine 27
Slide 28: Unmatched Patent Position in CRISPR Gene Editing
Unmatched Patent Position in CRISPR Gene Editing Cas9 Cpf1 Exclusive access to Cas9 and Cpf1 patent portfolios, which are independent of each other Exclusive access to advanced forms including high specificity, PAM variants, others High- Designed Engineered Variants for Engineered specificity PAM co
Slide 29: CRISPR Unlocks Genome Editing
CRISPR Unlocks Genome Editing Nuclease Complex of nuclease and guide RNA Cut precisely locates DNA Sites and cuts genomic sites eS PAM HiFi Ability to target Guide RNA multiple sites simultaneously Guide Sequence Nuclease can be engineered to reach more sites and to modulate cutting © 2018 2019 Edit
Slide 30: CRISPR Addresses Diverse Mutations
CRISPR Addresses Diverse Mutations Cut and Cut and Cut and Cut and Disrupt Remove Replace Insert Mutation Mutation Mutation PAM Insertion or Deletion Deletion Copy Insertion Homology-directed repair and Non-homologous end joining typically targeted insertion aim to disrupts a gene or eliminates prom