By John Lauerman and Michelle Cortez
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.
It’s also a cautionary tale of a long struggle to transform a scientific breakthrough into a treatment, and a lesson for companies working with today’s most talked-about medical technologies, such as the gene-editing tool Crispr.
“Expect a long haul,” Sharp said in an interview.
When RNAi pioneers Craig Mello and Andrew Fire won the Nobel Prize in 2006, champagne flowed, analysts predicted drugs for cancer and other disorders would rake in billions of dollars in sales, and dozens of companies jumped into the fray. Most of them abandoned their efforts over the following decade.
