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Spirogen Ltd.

Executive Summary

UK start-up Spirogen Ltd. is developing novel DNA-targeting drugs to treat cancer.

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Roche Signs Up Ipsen--Again

Roche's October 2003 in-licensing of Ipsen's Phase I GLP-1 analog buys the Swiss group an initial stake in the fast-growing diabetes market. Most established diabetes players already have a compound in this class, but the partners hope that Ipsen's expertise in peptide delivery will set this program apart from competitors'. As the second licensing agreement between Roche and Ipsen in less than a year, the deal further validates Ipsen's pipeline, and suggests that Roche's carefully-managed partnership strategy continues to encourage repeat business.

RNAi-Based Drug Development

Two issues dominate discussions over the business prospects for RNA interference-based therapeutics: the first is a basic pharmacological challenge: can a double-stranded RNAi be delivered inside a cell, and can it survive entry through the cell membrane with the physical integrity necessary to bind its target RNA and shut down protein production before it starts? The second is determining who has freedom to operate, in an area where patents enabling human therapeutic uses for RNAi are relatively fresh, and clinical data does not exist.

RNAi Revs Up

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.

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