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Tucson, Arizona | Published: 10.03.2006
STANFORD, Calif. — The technology at the heart of Monday's Nobel Prize in medicine spawned a niche biotechnology industry almost as soon as it was discovered in 1997.
Today, three companies are testing experimental drugs on people, and several more are getting ready to move their research in HIV, influenza, hepatitis and other diseases from laboratory animals to humans.
At least a half-dozen biotechnology companies are developing drugs that silence genes by interfering with the messenger-carrying RNA, a technique discovered by the newest Nobel laureates, Andrew Fire of Stanford University and Craig Mello at the University of Massachusetts. There are eight U.S and European patents specifically related to the technology.
The Nobel was awarded to the two scientists for discovering how to silence mutant genes through RNA, groundbreaking work that expanded scientists' horizons beyond human genes.
RNA was originally viewed simply as a passive messenger that took DNA's recipe from the cell's nucleus and delivered it to protein-building machines called ribosomes.
Many drug makers and researchers are betting RNA interference will be a powerful tool in customizing drugs.
The idea, essentially, is to mug the messenger RNA before it can deliver its genetic information, thereby "silencing" genes.
Last year, small Horsham, Pa.-based Nucleonics Inc. received nearly $50 million to begin human tests of its experimental hepatitis drugs, once it receives Food and Drug Administration approval.
Sirna Therapeutics Inc. is conducting human tests of its drug to treat the eye disorder macular degeneration, and Alnylam Pharmaceuticals is also testing its experimental medicine to treat a respiratory disease on people.
But Fire cautioned Monday not to expect any "miracle medicines" soon. He said it could be many years before his work pays off in a federally approved drug; other major biological advances have taken 20 years or more to bear fruit, he said. Even the most optimistic estimates predict that drugs won't reach the market until 2012.
The first applications for the discovery by Fire and Mello will be in aiding scientists and doctors in their quest to find and combat disease-causing genes, Fire said.
"It will help physicians guide treatment by better understanding disease," he said in a news conference at Stanford.
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