Mirus granted European patent for gene therapy

Madison, Wis. - Mirus Bio Corp., a Madison-based biotechnology firm, has won a European patent, its first outside the U.S., that covers a method of treating patients with muscle and other tissue diseases with an injection of genes.

The patent, titled "Intravascular Delivery of Nucleic Acid," covers a method for delivering a wide range of therapeutic nucleic acids to body tissues via the blood stream and compliments a patent already obtained in the United States.

The delivery technique can be used in clinical applications of gene therapy methods for treatment of muscular dystrophy, peripheral vascular disease, and Lou Gehrig's disease, also known as amyotrophic lateral sclerosis.

Patients with the most common muscular dystrophies, Duchenne and Becker, suffer from a genetic defect in which dystrophin protein is either poor or inadequate in their muscle cells. It affects roughly one of every 3,500 male births, according to Mirus.

The Mirus technique works by injecting a pDNA (plasmid DeoxyriboNucleic Acid) saline solution intravenously, where it is absorbed into adjoining muscle cells. Once the nucleic acids are inside the muscle cells, they naturally produce the encoded therapeutic agent. "What we're doing when we're putting [nucleic acids] in intravenously is we can get the product into many, many, many cells throughout the limb," said Russell Smestad, president of Mirus. "It's a step toward an eventual cure."

Removing impediments

Smestad said that the dearth of safe and effective delivery methods has been the primary impediment to the development of gene therapies. He said investigators are increasingly recognizing the versatility of Mirus' intravascular delivery method and its ability to be scaled from research to clinical applications.

He added that Mirus is actively seeking strategic alliances to expand its product pipeline.

Mirus has 30 international distribtion centers, and its lead therapeutic for muscular dystrophy is being developed collaboratively with Transgene S.A. of Strasbourg, France. Smestad said the patent will benefit the French program while it strengthens the company's overall international business position.

"We have other aspirations [that] we will certainly be pursuing, relationships with other parties for other products," Smestad said.

The patent provides Mirus with intellectual property coverage throughout Europe, which is the largest pharmaceuticals market besides the United States. The first clinical study of the technique will probably begin in 2008, Smestad said.

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