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Transposagen Biopharmaceutical’s 2012 current corporate overview of our translational RandD + preclinical services.
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© Transposagen Biopharmaceutics, Inc. 1
Better Models. Better Results™
Genetic Modification for Better Disease Models™
© Transposagen Biopharmaceutics, Inc.
About Us
Technologies: • piggyBac™ •XTN™ TAL Target-Specific Nucleases •Rodent Stem Cells
Transposagen Biopharmaceuticals, Inc., is dedicated to providing better disease models through genetic modification. Transposagen specializes in custom and off the shelf XTN™ TAL Nucleases, animal models, cell lines, stem cells and cutting-edge research tools and technologies to improve drug discovery and development research. These unique technologies and services for genetic modification give customers and technology users access to any genetic modification in any gene in any genome for both research and biologic production purposes.
Applications: •Rodent Model Creation •Cell Line Engineering & Stem Cells •Genetically Engineered Plants •Biologics Production •Therapeutics
© Transposagen Biopharmaceutics, Inc.
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.
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.
A. Site specific binding and induced dsDNA breaks
B. Transfect SSCs
C. Nested PCR confirmation of Homologous Recombination events
Site Specific Knock-in
© Transposagen Biopharmaceutics, Inc.
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.
piggyBac vs. Viral Vectors
• Cargo capacity greater than 120 kb • Delivery of entire BACs reported
Characteristic Viral Vectors piggyBac
Safety
Induced Immune Response
Oncogene Activation
Production Costs
Cargo Capacity
X
X
X
X
X
Source: Current Gene Therapy (2004), 4, 357-372
1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20
Cargo Capacity (kb)
piggyBac
Lentivirus
Retrovirus
Adenoassociated Virus
120 kb
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piggyBac™ Cumate Inducible Switch Titratable and can turn back off easily
© Transposagen Biopharmaceutics, Inc.
Reversible Transposition Pop-out the transposon
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Targeted Integration and Excision
• Gene Therapy • Cell Lines
Animal Models Therapeutics Cell Lines Bradley et al Nature (2011) In Press
© Transposagen Biopharmaceutics, Inc.
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.
Animal Models
• Knockout
• Transgenic Overexpression
• Conditional
• Knock-in
• Transgenic knockdown
• Humanized
Rat and mouse models available Any species possible
© Transposagen Biopharmaceutics, Inc.
Creation of Animal Models using SSCs
Figures Courtesy of Dr. Kent Hamra
© Transposagen Biopharmaceutics, Inc.
Genetic Modification Technology Comparison Primary Technology
Virus ZFN & HR Plasmid piggyBac & XTN
High Efficiency Yes No No Yes
Non-viral safety No Yes Yes Yes
Integration control
No Yes No Yes
Unlimited DNA Cargo size
No No No Yes
Reversible footprint free
No No No Yes
Site specific gene editing
No Yes No Yes
Cell lines and Animal Models
Yes Yes No Yes
Licensing restrictions
No Yes No No
© Transposagen Biopharmaceutics, Inc.
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.
Contact Information
Jack Crawford, MS Director, Business Development [email protected]
