Transposagen Expands Field of Use for piggyBac Technology

Editor's note: Transposagen Biopharmaceuticals Inc. is located in UK's on-campus incubator ASTeCC.

LEXINGTON, KY (Oct. 17, 2009) − Transposagen Biopharmaceuticals, Inc. based in Lexington, has come to an agreement to expand its license for piggyBac technology to cover nearly all commercial applications. The intellectual property, owned jointly by the University of Notre Dame, the University of Florida and the United States Department of Agriculture, enables facile genetic manipulation of most species. It is the core technology used by Transposagen to create TKOTM Knockout Rat Models, laboratory rats with a single gene disruption that mimic human disease.

The laboratory of Dr. Malcolm J. Fraser, Jr. at the University of Notre Dame has been responsible for the early characterization and development of the piggyBac DNA transposon. PiggyBac is a highly versatile technology that is used for genetic engineering in almost any animal, allowing for both mutagenesis (changing or disrupting genes) and transgenesis (adding genes). PiggyBac is now enabling genetic manipulation for a wide range of important species including research animals and agriculturally important animals for which genetic manipulation was previously impossible or cost-prohibitive.

"Transposagen was already in the process of using piggyBac to generate tens of thousands of knockout rat lines in a very short period of time. We will now be able to use piggyBac to modify the genomes of other important organisms," said Dr. Eric Ostertag, CEO of Transposagen. "PiggyBac is also finding uses in human therapeutics as it can be used to re-program cells to become induced pluripotent stem (iPS) cells. In the long-term piggyBac may even be used for human gene therapy."

As part of the licensing deal, Transposagen will be responsible for distribution of piggyBac to researchers working in academia and other not-for-profit institutes and will also control commercial sublicenses. "We will now be able to provide piggyBac to pharmaceutical companies as a novel tool for drug and biomarker discovery," said Ostertag.

"This new license with Transposagen will greatly expand the commercial use of piggyBac," said Dr. Malcolm Fraser, professor in the Department of Biology at the University of Notre Dame.

Transposagen's new Vice President of Research, Joseph Ruiz, Ph.D., is a world expert in stem cell and iPS cell technology and will provide expertise in developing some of Transposagen's new laboratory models which incorporate iPS technology, including rat models with fully humanized organs. In addition to establishing an Animal Stem Cell Core at the Indiana University School of Medicine in 2002, Dr. Ruiz was most recently the Director of Research and Development at Vesta Therapeutics (Research Triangle Park), where he was responsible for developing protocols for manufacturing stem cell-derived hepatic (liver) cells for drug discovery and therapeutic applications.

"Transposagen and Notre Dame are staying on the cutting edge of new mobile DNA and non-embryonic stem cell technologies," said Dr. Ruiz. "Non-embryonic stem cell technologies share most of the therapeutic promise of embryonic stem cells, without having the ethical dilemmas. The piggyBac field of use expansion will ideally position Transposagen as a leader in this very promising new area."

About Transposagen Biopharmaceuticals
Transposagen Biopharmaceuticals, Inc., a Lexington based company, is the worldwide leader in the creation of unique genetically modified rat models. The company's signature product, TKOTM Knockout Rat Models, mimic human diseases and are used for drug discovery and development research. The production of animal models is a $1.2 billion/year market and is expected to grow 12% annually through 2012.