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Mirus Bio awarded patent on RNA delivery

Madison, Wis. - Mirus Bio Corp., a Madison-based company engaged in gene therapy and the study of RNA interference, has been granted a United States patent on a new injection delivery method.

The company has been working to address the lack of an effective method to deliver nucleic acids such as RNA for the study of gene function, and along with collaborators at the University of Wisconsin-Madison, it has developed a more rapid method of injection.

RNA interference is a cellular process in which nucleic acids, which convey genetic information, are delivered in short sequences and used to regulate - to suppress or silence - gene expression and protein production in animals and humans. The company believes the new delivery method, combined with RNA interference, creates a power new research tool. It is actively pursuing strategic alliances and licenses “to apply the technology as widely as possible,” according to chief executive Russell Smestad.

"There is nothing that I'm able to announce at this point in time, but we're actually in negotiations with other parties, and hopefully in the weeks and months ahead, we'll be able to announce something," Smestad said.

The new technology is an advancement of hydrodynamic injection, an established delivery method that conveys nucleic acids through the bloodstream to surrounding tissues and cells. In most cases, this standard injection of DNA or RNA into a vein or artery is enough to retain the nucleic acids in the blood vessel until it is degraded and filtered out of the body.
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Nucleic migration

In developing the new method, researchers found that rapid injection of a large volume of a nucleic-acid saline solution, if done while simultaneously altering the permeability of blood vessel walls - through mechanical or biological means - enables DNA and RNA to migrate beyond surrounding tissues. This enables regional delivery throughout an entire limb.

"In order for any gene-silencing method to work, one has to not only get them into the body, but you've got to get those compounds into the target cells, where they can do their function," Smestad noted. "We've got an interesting platform that no one else in the world has, so it's very much a cutting-edge technology."

Hydrodynamic injection has been widely adopted for in vivo, or inside the body delivery to the liver, where it serves as a tool for target identification and validation studies. In the future, Mirus Bio anticipates that a product based on the new delivery method will be used to deliver nucleic acids to skeletal muscle - creating a new platform for human therapeutics.

Dual capability

Although he described the injection method as a cutting-edge technology that gives Mirus Bio a dual capability with respect to gene expression and gene therapy, Smestad downplayed its impact on projected growth. Mirus Bio, established in 1995, employs 47 people in its University Research Park facility. In collaboration with Transgene S.A. of Strasbourg, France, the company already is developing a therapeutic for the treatment of muscular dystrophy.

"It doesn't have an immediate impact on growth," Smestad said of the new patent. "It's really a new capability that we've got, and our intent is to allocate more of our existing resources to this area, as compared to focusing strictly on gene therapy, which is the opposite of what we're doing here."

Gene therapy, he noted, involves the delivery of nucleic acid to "up-regulate" or increase gene expression. "What we're doing in this patent is delivering a different type of nucleic acid to down-regulate or suppress gene expression," he elaborated. "It gives us a real dual capability as a company to modulate that gene expression either up or down depending on what the objective might be."

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