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EXECUTIVE SUMMARY as of 2006
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With pharmaceutical pipelines running dry, companies are on the lookout for the next breakthrough technology that can lead to a pharmaceutical treatment. Is RNA interference (RNAi) that breakthrough?
For 50 years since the discovery of the DNA double helix, RNA has been a necessary but not too exciting participant in protein synthesis, but that all changed with the discovery that a specific type RNA could silence plant genes. The significance of the report was recognized when the silencing phenomenon was also observed in nematode worms and subsequently in a wide variety of cells including mammalian cells. These discoveries launched a new dimension to molecular biology and set astronomically high expectations for this new technology.
The purpose of this report is to assess the technology development status of RNAi and to determine which parts of the technology is ready for prime time. To complete the assessment, PI sifted through ~12000 patents related to RNAi research, disease or pharmaceutical applications. By categorizing the patents into business and technology relevant groups, Patent Insights was able to identify trends in technology development. Supplementing the patent trends with information from ~13000 journal references, PI was able to assess the significance of the patent trends and to make some educated predictions about the future direction of RNAi technology.
Will RNAi technology meet the market expectations? Our assessment suggests that the answer is yes and no.
1. RNA Interference as a pharmaceutical or as a research tools represents the junction between several technologically distinct areas.
2. RNAi technology is still new. Publication activity is exploding. Patenting activity has been exploding, but the rate of the explosion MAY be cooling off somewhat this year. IF the explosion is cooling, it signals a change.
3. About 2600 organizations and inventors participate in RNA Interference, but less than 100 organizations hold significant intellectual property portfolios, suggesting that RNAi is in an early technology growth phase. As growth slows, a shakeout might be expected. During the shakeout, partnering, licensing or acquisitions will be important.
4. Most of the key patents belong to universities and university spin-offs. In fact, in our RNAi database 50% of assignees are universities, 25% are companies and 25% are individual inventors. Also, 80% of the US or EP patents in the database are still applications.
5. It is clear that the scientific foundation for RNAi is being built at the same time the technology is being commercialized, producing significant risk of miss-steps by commercial organizations. Miss-steps cost both money and time, both precious commodities in product development.
6. Over half the patents in the RNAi database and more than half the documents in the RNAi literature database deal with expression analysis and the correlation of disease and gene function. With such a heavy existing focus, it is clear that RNAi technology is ready to support research functions such as correlating gene expression with disease state. This area is already well underway.
7. It is probable that RNAi technology can be used as a therapeutic when access to the delivery site involves already existing methods. For example, delivery of RNAi by injection into the eye is currently in clinical trails. While not in any known trials, stents represent another method to deliver RNAi reagents that should have a high-probability of success. In Patent Insights’ view, the idea of hooking RNA interference process with an easily accessible location and to an already well understood delivery method is a simple first step. Since few seem to be on this path, PI views this strategy as a technology white spot, an opportunity.
8. Until a safe, generally effective method can be found to deliver siRNA or miRNA to chosen cells, the promise of making RNAi therapeutics is unlikely to be fulfilled. Efforts toward this goal appear to be in an exploratory phase, and it is not yet clear which method will win in the end. However, transfection and pulmonary delivery methods are PI?s favorites for now.
9. Legal battles have not yet broken out in the RNAi community. In part, we believe that it is a lull before a storm. In part, we can admire the intellectual property aggregation strategy of Anylam.
THE BOTTOM LINE as of 2006
As a result of our study, PI believes that RNAi technology can grow as predicted to support the R&D community.
PI also believes that a ?white-spot? opportunity exists to provide RNAi technology for accessible disease conditions using existing devices and delivery methods.
Because tried and validated delivery methods for RNAi do not yet exist, PI believes that some of the predictions of high double-digit growth in pharmaceutical applications are probably over stated and it seems unlikely (in our opinion) that such rapid growth of RNAi as a pharmaceutical can be supported by technology we can see today.
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