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January 13, 2003 

“Sleeping Beauty” Technology Holds Promise of Breakthroughs in Gene Therapy

MINNEAPOLIS, MN — A U.S. patent has been issued for a synthetic DNA that will allow scientists to conduct gene therapy without injecting viruses that may pose significant risks to humans. This invention, licensed exclusively to Discovery Genomics Inc. (DGI) an early-stage Minnesota biotechnology company, has been issued to the University of Minnesota.

The Sleeping Beauty Transposon™ System is a leading long-lasting way to insert genes into the chromosomes of the cells without using a virus. Gene therapy uses a patient’s cells to manufacture proteins that will correct either an inborn genetic disorder or an acquired disease, such as cancer, by inserting a gene into the patient’s chromosomes.

"In the next decade, genetic engineering of human cells will be common," Perry B. Hackett, Ph.D., DGI’s Chief Science Officer, said in a Nov. 15 Genetic Engineering News cover story.

One of the biggest problems using viruses to deliver genes to cells in humans is the natural immune response the human body has to fight infections. Large doses of viruses can lead to immunologic shock and in extreme cases, death.

Sleeping Beauty technology grew from the discoveries Hackett, a molecular biologist at the University of Minnesota, made in his laboratory. Hackett’s group discovered an extinct gene that had existed in fish 15 million years ago, "awakened" the gene and modified it to work as a transfer agent for other genes.

The SB system Hackett developed, dubbed "Sleeping Beauty," improves upon existing gene therapy methods because it consists only of DNA, unlike viruses that are far more complex. The synthetic DNA carries the gene the same way a truck carries a load. The gene therapy agent DGI developed can place its “gene” load into a chromosome. The transferred gene then operates in place of a defective gene to cure a disease.

Sleeping Beauty:
Provides an efficient way to deliver genes into the chromosomes of humans and animals for treatment or prevention of diseases and disabilities.
Gives researchers the ability to introduce therapeutic genes directly into chromosomes, where they can provide long-lasting therapy.
Accommodates genes of many sizes, which means more diseases and disorders can be treated with it than with conventional virus-driven therapy.

John E. Haaland, President and Chief Executive Officer for DGI, said the patent is “a major milestone in DGI’s growing patent portfolio.”

“It positions us as a leader in gene therapy initiatives and will be a significant factor in our long-term growth,” Haaland said.

The next steps in research are to find an effective and safe method to target cells of specific tissues and to select specific genetic diseases for clinical trials.

Therapeutic targets for DGI include cystic fibrosis, lung cancer, acute lung injury, lysosomal storage disease and hemophilia. The Sleeping Beauty Transposon System is available for testing of other genes for other diseases and disorders.

The University of Minnesota received U.S. Patent 6,489,458 on Dec. 3 for a DNA-based transposon system for the introduction of nucleic acid into the DNA of a cell. The patent is exclusively licensed to DGI, which plans to commercialize the technology.

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Discovery Genomics is an early stage functional genomics company created to rapidly discover gene functions, conduct drug target validations, and design therapeutic agents based on genomics information. DGI, formed in October 2000 and based in Minneapolis, MN, has exclusive licenses to MorphantSM Technology and the Sleeping Beauty Transposon™ System from the University of Minnesota. Statements in this press release that are not strictly historical are "forward looking" statements as defined in the Private Securities Litigation Reform Act of 1995. The actual results may differ from those projected in the forward-looking statement due to risks and uncertainties that exist in operations and business environment.


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