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Mar 15, 2011 (Vol. 31, No. 6)

GEN's 30th Anniversary: Antisense

  • Editor's Note

    Recent news stories have noted that RNAi technology, once thought so promising among many in the life science community and especially big pharma, is now falling out of favor. The biggest hit took place last November when Roche dropped out of RNAi R&D after plunking down a half a billion bucks to explore the field over a four-year period. Delivery issues and the lack of a sooner-than-later expected payback helped lead to the Roche decision.

    In the meantime, industry reports point out that large pharma firms have been in-licensing antisense technology, which was developed over 20 years ago. What’s ironic is that antisense, just like RNAi, is intended to shut down the activity of specific genes. And similar to RNAi, antisense has had its own share of delivery problems.

    But unlike RNAi, antisense has a long history of trial-and-error and experimentation with lessons learned along the way. That’s probably why some biotech and pharma firms are giving it another closer look.

    This issue’s article commemorating GEN’s 30th anniversary is reprinted from November/December 1988. It is very timely in that it provides one of the first in-depth profiles of antisense technology and its range of potential applications.

    This story, like all the old stories that we are reprinting, demonstrates a particular significance and relevance for the life science research community.

    —John Sterling, Editor in Chief

  • "As Seen in GEN—Flashback" Volume 8, Number 10 November/December 1988

    Click Image To Enlarge +

    Researchers Pursue “Anti-Sense” Technology In Quest for Novel Drugs and Agriproducts

    By Anne Simon Moffat

    More flavorful fruits and vegetables, new treatments for a range of genetic diseases and a cure for AIDS are possible prizes for researchers pursuing a new technology, known as “anti-sense agents.”

    “If anti-sense approaches work, they will usher in a new era of drug therapy, and a new wave of [pharmaceutical] companies,” says Dr. Douglas Melton of Harvard University and a pioneer in the field. He believes that anti-sense approaches may offer our best hope for curing AIDS.

    “During the past two years, progress in anti-sense research has been asymptotically vertical,” says Michael Riordan, president of Gilead Sciences, Inc. (Foster City, CA), one of two U.S. companies dedicated to the development of anti-sense agents. The worldwide market for new products that stem from anti-sense research has been estimated to be in the range of $2 to $20 billion annually.

    Anti-sense agents block gene function. It has been known for about 10 years that anti-sense mechanisms can control gene expression in bacteria. But within the past few years, it has been shown that such agents may also be manipulated in eukaryotes to inhibit the action of “bad genes”, such as those that code for an enzyme that accelerates food spoilage or those needed for virus replication.

    However, no one has proven that anti-sense agents can perform in man. Still, because many human diseases can be attributed to a single deleterious gene, researchers are hopeful that anti-sense agents can be used therapeutically, to clamp down on problem genes.


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