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Editas Medicine Corporate Presentation Corporate Presentation January 13, 2020 PIONEERING THE POSSIBLE editasmedicine.com © 2020 Editas Medicine 1 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 regarding the Company’s strategy, future operations, future financial position, future revenue, projected costs, prospects, plans, and objectives of management, are forward-looking statements. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this presentation include statements regarding the clinical trial timelines for EDIT-101 (AGN-151587) and EDIT-301. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; and other factors discussed in the “Risk Factors” section of the Company’s most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this presentation represent the Company’s views as of the date of this presentation. The Company anticipates that subsequent events and developments will cause its views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this presentation. © 2020 Editas Medicine 2 Building a Genomic Medicine Leader CRISPR Gene Editing to Develop Differentiated, Transformational Medicines for High Unmet Need In Vivo CRISPR Medicines Engineered Cell Medicines Develop best-in-class medicines for Leverage AAV-mediated editing with SaCas9 hemoglobinopathies using Cas12a and solid into additional therapeutic areas tumors using iPSC-derived cells Maintain Best-in-class Platform & Intellectual Property, and Advance Organizational Excellence CRISPR: clustered regularly interspaced short palindromic repeat; SaCas9: Staphylococcus aureus CRISPR-associated protein 9; Cas12a: CRISPR-associated protein 12a; iPSC: induced pluripotent stem cell © 2020 Editas Medicine 3 2019 Achievements In Vivo CRISPR Medicines Engineered Cell Medicines Initiated first ever clinical trial of an in vivo CRISPR medicine Initiated IND-enabling activities and presented preclinical data with EDIT-101 for Leber congenital amaurosis 10 (LCA10) for EDIT-301, for sickle cell disease and β-thalassemia Achieved in vivo preclinical proof-of-concept and declared EDIT- CANCER FOCUS 102 development candidate for Usher syndrome 2A (USH2A) Focused collaboration with Bristol-Myers Squibb on αβ Advanced autosomal dominant retinitis pigmentosa 4 (RP4) T cell medicines program Advanced engineered iPSC-derived NK (iNK) cell medicine Expanded into neurological diseases in partnership with AskBio for solid tumors using technology from BlueRock Therapeutics Dose first EDIT-101 patient Generated edited NK cells from healthy donors and iPSCs with significantly increased anti-cancer activity Maintain Best-in-class Platform & Intellectual Property, and Advance Organizational Excellence Hired Chief Executive Officer, Grew team to 196 Editors Added $75M through business Chief Medical Officer, Chief Financial Officer, development activity and Senior Vice President of Operations NK: natural killer © 2020 Editas Medicine 4 Pipeline PROGRAM (OR DISCOVERY LEAD IND ENABLING EARLY-STAGE LATE-STAGE PARTNER STRUCTURE DISEASE/CANDIDATE) OPTIMIZATION CLINICAL CLINICAL IN VIVO CRISPR MEDICINES OCULAR EDIT-101 (AGN-151587): Partnered Leber Congenital Amaurosis 10 EDIT-102: Usher Syndrome 2A Collaboration Autosomal Dominant Retinitis Collaboration Pigmentosa 4 OTHER ORGANS Duchenne Muscular Dystrophy Wholly-owned (Muscle) Neurological Diseases Collaboration ENGINEERED CELL MEDICINES HEMATOLOGY EDIT-301: Wholly-owned Sickle Cell Disease β-Thalassemia Wholly-owned CANCER Healthy Donor NK Cells Collaboration iPSC NK Cells Wholly-owned / Collaboration T Cells Wholly-owned αβ T Cells Collaboration © 2020 Editas Medicine 5 2020 Priorities In Vivo CRISPR Medicines Engineered Cell Medicines Dose first EDIT-101 patient in Q1 File EDIT-301 IND for sickle cell disease EDIT -101: complete adult low and mid-dose cohorts CANCER FOCUS by year-end Initiate IND-enabling studies for an engineered healthy Achieve in vivo preclinical proof-of-concept for a donor NK (HDNK) cell medicine to treat solid tumors neurological indication Achieve in vivo preclinical proof-of-concept for an Nominate development candidate for RP4 engineered iNK cell medicine to treat solid tumors Advance αβ T cell medicines in collaboration with Bristol-Myers Squibb Maintain Best-in-class Platform & Intellectual Property, and Advance Organizational Excellence Build out clinical and medical affairs organization Advance manufacturing and operations to support clinical activity © 2020 Editas Medicine 6 In Vivo CRISPR Medicines editasmedicine.com © 2020 Editas Medicine 7 Targeting Genetic Blindness EDIT-101 Remove genetic mutation to restore CEP290 protein and rebuild photoreceptors in patients with Leber congenital amaurosis 10 DISEASE EPIDEMIOLOGY PROGRAM STATUS Degeneration of 2-5K First patient photoreceptors leading to patients in US dosing expected in Q1 2020 blindness in childhood and Europe with potential for data this year CEP290: centrosomal protein 290 © 2020 Editas Medicine 8 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 © 2020 Editas Medicine 9 EDIT-101 Demonstrates Rapid Onset of Therapeutic Editing CEP290 GENE EDITING MOUSE FULL-THICKNESS RETINA 100% 100% 1E+13 vg/mL n = 10 mice EDIT-101 1E+12 vg/mL n = 8 mice n = 13-75 mice 80 60 log scale log 10 – 40 Editing Editing Transduced Region Transduced 20 Productive Editing in in Editing Productive Therapeutic Threshold 1 0 1 6 11 16 21 26 100 1010 1011 1012 Week post dosing Vector Dose (vg/mL) Rapid, therapeutically relevant editing at AAV dose that has been safely administered to humans AAV: adeno-associated virus Maeder et al. Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10. Nat Med. 2019 Jan 21; 25:229-233 © 2020 Editas Medicine 10 First In Vivo CRISPR Clinical Trial Initiated LCA10 PHASE 1/2 STATUS Enrolling CLINICAL TRIAL PATIENTS Approximately 18 patients, aged 3 years and above INTERVENTION Single dose of EDIT-101 administered via subretinal injection to eye with worse vision Open-label, dose escalation CONTROL Patient’s own baseline value for each efficacy measure study to evaluate safety, ENDPOINTS • Primary: Safety including frequency and number of adverse tolerability, and efficacy of events related to drug, procedure, and dose limiting toxicities EDIT-101 (AGN-151587) in • Secondary: Efficacy including visual acuity, mobility course, patients with CEP290 IVS26 macula thickness, pupillometry, and electroretinogram mutation* FOLLOW-UP Core measurements every 3 months for 1st year *Intervening sequence 26 in CEP290 gene containing the c.2991+1655A>G mutation © 2020 Editas Medicine 11 EDIT-102 to Treat Usher Syndrome 2A EDIT-102 Rescue vision by restoring USH2A protein leveraging same proprietary SaCas9 enzyme, vector, and promoter as EDIT-101 DISEASE EPIDEMIOLOGY PROGRAM STATUS Progressive vision loss 4K EDIT-102 leading to blindness due patients with target development candidate to degeneration of mutation declared photoreceptors Additional 10K potentially addressable © 2020 Editas Medicine 12 EDIT-102: Editing Restores Functional Usherin Protein increases USH2A mRNA and restores Deleting exon 13 Message box lackingMessage exon box 13 functionalMessage protein box human cell line at 4 days human retinal organoids at 120-140 days USH2A Gene Productive Editing ∆13 USH2A mRNA c.2299delG 100% 100% Healthy retina Ex12 *Ex13 Ex14 USH2A retina Gene editing to remove exon13 61% 47% Edited Ex12 Ex14 0% 1% 0%0 0%0 USH2A retina Unedited USH2AEdited Unedited USH2AEdited Edited Edited Usherin Protein Complex
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