SBIR and STTR Success Story for
Targeting Systems

(Information Posted/Updated on 05/18/2007)

Targeting Systems
8605 Fiona Way
Santee, CA  92071

Contact:    Dr Rampyari Walia
Phone:      619-562-1518
Fax:          619-562-1326
Web Site:

Related Award(s):   R43 DK51466-01
Technology Developed:
Novel technology for high throughput screening assays for drug discovery, Transfection reagents for human stem cells, human endothelial cells, hepatocytes and many hard-to transfect primary cell types, reagents for efficient intracellular delivery of functionally active proteins and peptides, and novel luciferase technology including vector systems and luciferase assay reagents for applications in functional genomics. One of these novel luciferases, Gaussia luciferase, is a secreted luciferase over 1000-times brighter than firefly and renilla luciferase, Reagents and kits for gene silencing and siRNA screening. Technology for non-invasive imaging and tracking of implanted human stem cells using bioluminescent imaging techniques, quantitative assessment of stem cell survival and expansion in vivo.

Key Words:  High throughput screening, Transfection, Protein delivery, siRNA delivery/screening, Luciferase assays/vectors, Lentiviral vectors, Stem cell tracking, Stem cell transfections.
Uses of Technology/Products/Service:
The Targefect reagents (for gene transfer and siRNA delivery) have been widely used and are now cited in approximately 100 citations in reputable journals. The Profect reagents (for protein delivery) have also been commercialized and are cited in 8 citations.

Modifications of the Targefect and Profect reagents have proven useful for efficient gene and protein delivery into human stem cells. Gaussia luciferase based lentiviral vectors have proved extremely useful for tracking stem cells and monitoring stem cell survival and expansion in vivo. This technology has now been transferred to our recent spin-off Pluristem Innovations.

The novel luciferase technology developed at Targeting Systems is now being used for studying reporter gene expression and for HTS (high throughput screening) in drug discovery applications.

Benefit to Company:
Targeting Systems started as a one-person operation entirely with the help of an NIH SBIR Phase I grant. The company commercialized its first line of products, the Targefect reagents and became profitable within 1 year of inception.

Revenues from the Targefect reagents in subsequent years helped us to develop new technologies for protein delivery, gene silencing and chemiluminescence-based applications in functional genomics and drug discovery. In addition we were able to spin off a new company "Pluristem Innovations" which offers novel technology for genetically engineering and tracking stem cells in January 2007.

How Product Was Commercialized:
The products were commercialized mostly by word of mouth and press releases in the New Products section of Bitoech publications -Biotechniques, Genetic Engineering News, and Science and by Google based advertising.

Other Comments Related to Company's Success Story:
Just having one SBIR grant not only enabled us to establish a successful company with active R&D, spin off a second company and also enabled us to reach out to the local community. Targeting Systems participated in providing Biotech internships to some of the local high school students and one of the projects conducted under this program won 5 awards at the local Science fair.

Another tremendous advantage of SBIR grants is that they enable young investigators to conduct active research funded by revenues from their startups. Without the SBIR system it is very hard for young scientists in academia to start a company without going in for venture capital.

Past R&D and/or Sales from this Project:   $1 million
Estimated Future Annual R&D and/or Sales from this Project:   $3 million