Back in 2006, when the Nobel prize was awarded for the discovery of RNA interference, many companies hoped that the future of pharmaceuticals might lie in the ability to silence certain genes using RNAi. This process works fairly well in the lab, but it proved so difficult to make it work in an actual human that many companies gave up on the concept.
However, Alnylam Pharmaceuticals has come out with an RNAi drug that has been shown to be effective in humans. This is the first time RNAi has ever been shown to work outside of the lab. While the drug is still in its earliest stage of testing, the drug made a statistically significant difference on patients who took high doses of it. The disease used in testing is TTR-mediated amyloidosis, which basically causes a buildup of proteins that causes nerve damage. The drug aimed to silence the TTR gene, and was delivered into the body in lipid packets, which were carried to the liver, where TTR genes are expressed.
Even if this particular drug doesn't prove to be completely effective, this breakthrough is exciting because it shows that RNAi drugs probably can work in humans.