1. February 11, 2016
Vic Myer, Chief Technology Officer

2. The Potential to Repair Any Broken Gene
Confluence of human genetics, delivery technologies, and CRISPR genome editing
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
© 2016 Editas Medicine

3. Scalable Platform Targeting CRISPR Medicines
Wide range of technologies underpinning translation of the technology
Achieve Right Repair
Specifically Edit Wide Range of Mutations
 New Cas9 species and variants
 Engineered Protein and gRNAs
 Identification of Lead Molecules
 Cut orientation
 Donor template engineering
 Exogenous repair factors
Directed Editing
Nuclease Engineering
Control & Specificity
Delivery
Tightly Control Cutting
Reach Site of Disease
 Multiple orthogonal specificity
 All-in-one AAVs
 Electroporation of RNA/RNP
 Lipid nanoparticles
measurement technologies
 Tissue selective promoters
 Self-regulating systems
© 2016 Editas Medicine

4. Biology, Clinical, Regulatory
Multiple Potential Therapeutic Areas
~6,000 diseases caused by genetic mutations; ~95% have no approved therapy
 Repair mechanism required for edi4ng
 Presence of common muta4on or mul4ple muta4ons
 Ability to iden4fy specific and efficacious leads
 Needs not presently being adequately addressed by exis4ng therapeu4c modali4es
 Ability to generate a differen4ated product
Medical Need
Priority Disease Indica0ons
Biology, Clinical, Regulatory
Technology
 Number of cells and type of cells to target
 Demonstrated methods for specific target cell and 4ssue type
 Disease natural history and ability to affect disease with edi4ng
 Sufficient clinical endpoints and access to pa4ents to carry out clinical trials
Delivery
© 2016 Editas Medicine

5. Diverse Pipeline Across Range of Diseases
Targeting first product candidate in clinic in 2017 with additional programs to follow
Target
Editing
Delivery
Commercial
IND
Our Programs
Gene
Mechanism
Mode
Rights
Discovery
Enabling
Phase I
Eye Diseases
NHEJ – Small Deletion
Leber Congenital Amaurosis 10
CEP290
AAV in vivo
2016
2017
Genetic and Infectious Disease(s) of Eye
Examples: Usher Syndrome 2a, HSV-1
Multiple
NHEJ
AAV in vivo
Engineered T Cells
Juno
Gene Editing in T Cells to Treat Cancer
Multiple
NHEJ
ex vivo
Additional Research Programs
Non-Malignant Hematologic Diseases
Examples: Beta Thalassemia, Sickle Cell
Multiple
NHEJ & HDR
ex vivo
Genetic Disease(s) of Muscle
Example: Duchenne Muscular Dystrophy
NHEJ – Small
& Large Del.
Multiple
Multiple
Genetic Disease(s) of Lung
Example: Cystic Fibrosis
Multiple
NHEJ & HDR
Multiple
Genetic and Infectious Disease(s) of Liver Example: Alpha-1 Antitrypsin Deficiency
NHEJ: Non-homologous end joining
Multiple
NHEJ & HDR
Multiple
HDR: Homology directed repair
© 2016 Editas Medicine

6. Potential Regulatory Framework for IND Filing
Product candidates that create directed, durable changes to the somatic genome are in the clinic:
 HIV and hemophilia (ZFN) and CLL and ALL (TALENS)
The procedures and standards applied to gene therapy products and cell therapy products may be applied to any CRISPR/Cas9 product candidates
 Within the FDA’s CBER, Office of Cellular, Tissue and Gene Therapies; Advisory Committee to advise CBER on its reviews
 CBER works closely with the NIH and their Recombinant DNA Advisory Committee (RAC), which makes recommendations to the NIH on gene therapy issues
Engages in a public discussion of scientific, safety, ethical, and societal issues related to proposed and ongoing gene therapy protocols.
Close working partnership with global regulators is critical
© 2016 Editas Medicine

7. M E D I C I N E