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Intronn Aims to Cure Diseases by Repairing Genetic Defects

By Michael S. Rosenwald
Washington Post Staff Writer
Monday, January 10, 2005; Page E05

The scientists working in Intronn's labs are trying to mend nature's mistakes.

If the work is successful, they will trick mutated genes into working properly, potentially treating hemophilia, increasing the body's "good" cholesterol or providing therapies for genetic diseases.

Executives and scientists of the genetic engineering firm Intronn at their laboratory in Gaithersburg. Gerard J. McGarrity, center, stands with senior scientists Colette Cote, left, and S. Gary Mansfield, finance and administration vice president Nancy Streicher, and senior scientist Madaiah Puttaraju. (Ricky Carioti -- The Washington Post)

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So far, that's a big if. Gaithersburg-based Intronn is one of several dozen biotech start-ups in the Interstate 270 technology corridor, mixed in among larger, revenue-producing firms such as Digene Corp., its neighbor across the street.

Intronn has about 15 employees. It has raised about $15 million and plans to soon ask venture capitalists for $3 million more to help move the research into human testing, possibly next year.

Intronn, formed eight years ago in North Carolina by two scientists, hired Gaithersburg resident Gerard J. McGarrity, a biotechnology industry veteran, in September 2000. He was the company's fourth employee.

To bring the company closer to his home and network of biotech associates, McGarrity said, he moved Intronn to a technology incbator in Maryland in January 2002. Intronn left the incubator little more than a year later for its current home.

The company works with genes, the bridge in the genetic process that starts with DNA. Genes make RNA, which in turn produce proteins, the foundation of cells and organs. Sometimes genes go haywire, resulting in proteins that can cause disease.

In conventional gene therapy, still in the experimental phase, normal genes are grown in labs and infused into the body to replace bad ones. The process is tricky, in part because the good genes can regrow in unintended places.

Instead of inserting new genes into the body, Intronn is working on what McGarrity calls "genetic Wite-Out." Using its patented molecules, he said, Intronn can splice correct instructions into RNA to repair the genetic defect.

"We can retype in our own message," McGarrity said. "We go into what's mutated and splice in the correct message."

But Intronn is at least several years from selling therapies based on the research. So far, the technology has spawned publication of 25 research papers in influential journals such as Nature Biotechnology and the Journal of Clinical Investigation.

Intronn's hemophilia program is the furthest along, and McGarrity said he hopes initial human testing can begin next year. Two other treatment programs are not as mature -- one for Alpha-1 Antitrypsin Deficiency, a hereditary disease that causes liver and lung disease, and another aimed at increasing the body's HDL cholesterol.

Though it is one of Intronn's least-developed areas of focus, McGarrity said the cholesterol program has attracted significant interest from drug companies because treatments for cholesterol can generate billions of dollars in revenue.

He said he has been working with several large companies to form a partnership but declined to identify them. Asked how close he was to signing such a deal, McGarrity said: "You never want to say 'close' because some of these companies move at a geological pace."

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