This company is the first to bring Nobel-prize-winning RNAi treatments to consumers

Last August, Cambridge, Massachusetts–based Alnylam Pharmaceuticals became the first company to bring an RNA interference (RNAi) treatment to market, winning FDA approval for its drug Onpattro. The drug treats peripheral nerve damage and other symptoms in people with the rare and previously untreatable condition of hereditary amyloidosis, a disease that causes abnormal amyloid proteins to build up in the body’s nerves and organs and interfere with tissue and organ function. According to CEO John Maraganore, RNAi treatments target ailments further upstream than most existing medicines by “silencing” the production of disease-causing proteins—without altering a patient’s underlying DNA code, which can yield unintended effects. Onpattro, he says, is just the start: Alnylam has five RNAi-based drugs in the final phase of trials—including ones targeting high cholesterol (which affects 11% of all Americans) and hemophilia—and several more in development. In addition, last May, Alnylam reported results of a study that successfully delivered an RNAi drug into regions of the brain and spinal cord, demonstrating the therapy’s potential to treat neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and ALS. The company has also implemented an innovative pricing model that will refund the full cost of Onpattro (list price: $450,000) and all future drugs if they aren’t effective. Says Maraganore: “We feel that value is best reflected in outcomes.”