Scientists Cracked a Deadly DNA Puzzle. Then Came the Hard Part

In 2002, renowned scientist Phillip Sharp co-founded Alnylam Pharmaceuticals Inc. after learning about an intriguing discovery: Researchers had figured out how to “silence” biological messages sent by the genetic code in worms.

Sixteen years and about $2 billion in research spending later, Alnylam has turned the concept, called RNA interference, or RNAi, into a treatment for people with a rare and deadly genetic disease. It’s the first RNAi therapy ever approved, and a small triumph of humanity’s mastery of his own genetics.