The Nobel Prize in physiology or medicine was awarded earlier this week. It went to Andrew Fire of Stanford University and Craig Mello of the University of Massachusetts Medical School for their work in RNA interference (RNAi). This technology is relatively young by Nobel standards, with the acknowledged research being completed less than 10 years ago. RNAi works by targeting messenger RNA and preventing it from being translated into protein, thus "knocking down" specific gene products. It is similar to antisense technology developed a decade earlier, but has so far proven more robust and effective.
The biotechnology industry has been quick to recognize and adopt RNAi as a tool in drug development programs, and to explore its potential as a therapeutic entity itself. RNAi uses short double-stranded RNA (siRNA) to effect protein knockdown. Providers of synthetic RNA, the consumable product for this technology, have already been merged into larger industry suppliers over the last few years, with Dharmacon being merged with Fisher Scientific (NYSE: FSH ) and Ambion with Applied Biosystems (NYSE: ABI ) .
Other companies are exploring the use of siRNAs as potential drugs themselves. These include publicly traded Alnylam Pharmaceuticals (Nasdaq: ALNY ) and Sirna Therapeutics (Nasdaq: RNAI ) . The ability of RNAi to target and deplete specific gene products gives it potential as a drug against both infectious diseases and genetic disorders caused by over- or undesired protein production. Both Alnylam and Sirna have been able to line up collaborations with big pharmaceutical firms such as Merck (NYSE: MRK ) , Novartis (NYSE: NVS ) , and GlaxoSmithKline (NYSE: GSK ) . This speaks to the recognized therapeutic potential of RNAi.
There are still obstacles to be overcome before siRNA-based therapeutics reach the market -- in particular, systematic delivery of these inherently unstable drugs throughout the body -- but advances in product stability by chemical modification and drug delivery continue to be made.
While drugs based on RNAi knockdown technology may have the potential to become the next big thing in biotechnology, they are still a long, long way from becoming FDA-approved marketable entities. But it's never too soon to start keeping an eye on the future -- the Motley Fool Rule Breakers newsletter specializes in identifying promising emerging technology companies, and you can try it free for 30 days.
As an aside, hats off to Stanford University. Along with a share of the aforementioned prize in medicine or physiology, the faculty also scored in chemistry, as the Nobel committee recognized the work of Dr. Roger Kornberg "for his studies on the molecular basis of eukaryotic transcription."
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GlaxoSmithKline is aMotley Fool Income Investorselection, while Merck is a former selection of that service.
Fool contributor Ralph Casale spent more than two years of research on a project that RNAi made all but irrelevant. He owns shares in GlaxoSmithKline but holds no financial position in any other firm mentioned. He was at one time an employee of Applied Biosystems.The Motley Fool has adisclosure policy.