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.
RNA interference, or RNAi, is a natural process some organisms use to destroy invading viruses, by degrading the genetic message prior to the production of protein. In so doing, the function of a gene can be silenced, without tinkering directly with the gene itself.
Although known to basic researchers for some time as a straightforward and efficient method for quieting the effect of a gene (they use RNAi in the same manner as a...