Back when I was in college studying genetics, messenger RNA (mRNA) and transfer RNA (tRNA) was really the only well understood uses of RNA in our cells. Then in 1998, the classical scientific dogma for the uses of RNA was dramatically altered by Fire, Mello and their colleges when they published their discovery of previously unknown use of RNA… to silence genes (which later resulted in a Nobel Prize)!
Not only was it a fascinating research tool but the newly branded interference RNA (RNAi) was an ancient defence mechanism built into our cells to defend them against viral hijackers, which in turn had incredible therapeutic potential. Fast forward nearly a decade on and we’re now starting to see some of the fruits of the research into this space.
Currently only 10% of RNAi therapies are in clinical development but it’s forecasted that five new Phase II/III RNAi agents (in wet age-related macular degeneration, RSV infection and acute renal failure) will be available on the market by 2012. This is an incredible estimate if you consider that it will then only taken about 14 years to convert a revolutionary basic science discovery into a useful therapeutic. On average it typically takes a biotech/pharma company 8-12 years to develop a standard drug, RNAi’s therapeutic development has moved at breakneck speed!