Scientists have recently recognized an apparently fundamental cellular mechanism that may be a sort of ancient immune system. Called RNAi, for RNA interference, the process utilizes pieces of double-stranded RNA to prevent gene expression. RNAi has quickly become a powerful research tool, but its real promise lies in its potential to generate a brand-new class of highly specific medicines. Researchers have only just begun to explore how RNAi works in mammalian cells, so there's no telling if it actually will give rise to therapeutics. A growing number of companies are betting they'll be able to turn the fad into fortunes. Already, some of the challenges to commercialization are obvious: how to deliver the RNA, and who if anyone will control intellectual property rights. Some firms aim to directly deliver short sequences of RNA, and those who travel this route may confront challenges similar to those faced by antisense companies. Others are trying gene therapy approaches and will inherit all the problems of that field. As in any new field, bluster and blather are mixed with secrecy about who has what in terms of technology, money, and patents. The winner is likely to be the first company that can reduce concept to practice, and come up with a drug that works.

Most genomics, combinatorial chemistry, and other biotech service businesses have been slow to provide clients the anticipated value, either in targets or compounds. Meanwhile, their competitors have caught up, commoditizing the technologies of even the industry leaders. With this lesson in mind, a new group of start-ups, pursuing "validated target discovery" technologies and the possibility of much faster timelines to targets and compounds, is racing to snare high-value partnerships while their technologies still have unique value. The partnerships won't leave all the chemistry and preclinical work to the clients--the low-risk strategy platform companies used to pursue--but will instead pay enough money so that the new start-ups, like Rigel and Arcaris, can create fully-integrated drug discovery platforms capable of producing IND-stage products. These start-ups need to take on the added risk in order to generate the required upside and create the only kind of enduring, high-value intellectual property in the drug industry: products themselves.