Transposagen Biopharmaceutical’s 2012 current corporate overview of our translational RandD + preclinical services.

1. Better Models. Better Results™
Genetic Modification for Better
Disease Models™
1
© Transposagen Biopharmaceutics, Inc.

2. About Us
Transposagen Biopharmaceuticals, Inc., is
Technologies: dedicated to providing better disease
models through genetic modification.
• piggyBac™ Transposagen specializes in custom and off
•XTN™ TAL Target-Specific the shelf XTN™ TAL Nucleases, animal
models, cell lines, stem cells and cutting-
Nucleases edge research tools and technologies to
•Rodent Stem Cells improve drug discovery and development
research. These unique technologies and
services for genetic modification give
Applications: customers and technology users access to
•Rodent Model Creation any genetic modification in any gene in any
genome for both research and biologic
•Cell Line Engineering & Stem production purposes.
Cells
•Genetically Engineered Plants
•Biologics Production
•Therapeutics
© Transposagen Biopharmaceutics, Inc.

3. piggyBac Site Specific Genetic Engineering
• Not limited to TTAA sites
• Xanthamonas TAL nuclease (XTN: a.k.a TALENs)
• TAL DNA binding modules and FokI DNA-cleavage
domain
• Site specific knockout, knock-in of any gene, any
genome
• Automated synthesis of XTNs
• Remove transposon after gene editing
© Transposagen Biopharmaceutics, Inc.

4. XTN™ TAL Nuclease KO Efficiency
• Rag1 XTN target site amplified, cloned and sequenced
• 16% disruption at the target site: better than Zinc-finger Nuclease and
Meganuclease
• Disruption sequences are shown below with XTN target site highlighted
in grey
© Transposagen Biopharmaceutics, Inc.

5. Site Specific Knock-in
A. Site specific binding and induced dsDNA breaks
B. Transfect SSCs
C. Nested PCR confirmation of Homologous Recombination events
© Transposagen Biopharmaceutics, Inc.

6. The piggyBac™ Transposon System
Key Features
•Efficient transgenesis using transfection
•Titratable number of integrations
•Large cargo limit – 10 to 100kb
•Rapid identification of integration site
•Reversible transposition – no footprint
•All-in-one inducible vector
•Safe non-viral system
© Transposagen Biopharmaceutics, Inc.

7. piggyBac vs. Viral Vectors
Characteristic Viral Vectors piggyBac
Safety X 
Induced Immune Response X 
Oncogene Activation X 
Production Costs X 
Cargo Capacity X 
Cargo Capacity (kb)
Adenoassociated Virus
Retrovirus
Lentivirus
piggyBac
1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20
Source: Current Gene Therapy (2004), 4, 357-372 120 kb
• Cargo capacity greater than 120 kb
• Delivery of entire BACs reported
© Transposagen Biopharmaceutics, Inc.

8. piggyBac™ Cumate Inducible Switch
Titratable and can turn back off easily
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9. Reversible Transposition
Pop-out the transposon
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10. Targeted Integration and Excision
• Gene Therapy
• Cell Lines
Animal Models
Therapeutics
Cell Lines Bradley et al Nature (2011) In Press
© Transposagen Biopharmaceutics, Inc.

11. Therapeutic Applications
• Gene and Cell Therapy
• Immunotherapy
PB kills ovarian cancer cells of tumor xenografts in mice
PB/TK cells stained red Apoptotic cells stain green Merged cells are yellow
© Transposagen Biopharmaceutics, Inc.

12. Animal Models
• Knockout • Knock-in
• Transgenic • Transgenic knockdown
Overexpression • Humanized
• Conditional
Rat and mouse models available
Any species possible
© Transposagen Biopharmaceutics, Inc.

13. Creation of Animal Models using SSCs
Figures Courtesy of Dr. Kent Hamra
© Transposagen Biopharmaceutics, Inc.

14. Genetic Modification Technology Comparison
Primary Virus ZFN & HR Plasmid piggyBac & XTN
Technology
High Efficiency Yes No No Yes
Non-viral safety No Yes Yes Yes
Integration No Yes No Yes
control
Unlimited DNA No No No Yes
Cargo size
Reversible No No No Yes
footprint free
Site specific No Yes No Yes
gene editing
Cell lines and Yes Yes No Yes
Animal Models
Licensing No Yes No No
restrictions
© Transposagen Biopharmaceutics, Inc.

15. Custom Services Overview
• Gene editing with XTNs (talens), HR vector, piggyBac Transposon
 Site specific knockout, knock-in, edit and correction of any gene in
any genome
• Stable cell lines
 Overexpression, knockdown, reporter lines, iPS reprogramming, iPS
and ES differentiation and selection, therapeutic applications
• Animal models
 Rat and mouse model generation available, additional species
possible
© Transposagen Biopharmaceutics, Inc.

16. Contact Information
Jack Crawford, MS
Director, Business Development
jcrawford@transposagenbio.com
© Transposagen Biopharmaceutics, Inc.