Founded to pursue antisense, Gilead recognized that antisense was too long-term to support the continual financings they knew would be necessary to create a self-sustaining drug business. Thus Gilead began a series of transformations, eventually crafting an image for itself as a near-term antiviral company. Sidebar: Isis Stays Pure: As Gilead has shape-shifted into an antiviral player, Isis has continued to carry the antisense torch. And, says Stanley Crooke, MD, PhD, the company's CEO, Isis' develop
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In-licensing Phase II ulcerative colitis drug Kappaproct from Swedish biotech InDex Pharmaceuticals wasn't the major move observers have been waiting for from Serono; it certainly doesn't reduce the Swiss firm's dependence on Rebif. But the $35 million deal endorses InDex's localized approach to antisense therapy, a failure-ridden area that many firms have given up on.
The world is waking up to aptamers. With a few big deals and a small flock of products in the clinic, aptamers' boosters see the molecules largely replacing antibodies; they've got many of the advantages of both large and small molecules, plus the added advantage of speedy creation. But they still face major technological challenges-including high manufacturing costs, limited half-lives, and problems with intracellular targets. The three players whose strategies we profile here-Corgentech, Coley and Archemix--have all chosen to pursue opportunities with aptamers which avoid their biggest problems. But the solutions also limit these companies' abilities to fully exploit the aptamer platform.
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.