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Most Innovative Companies

Alnylam Pharmaceuticals

Alnylam became the first company to bring an RNA interference (RNAi) treatment to market, winning FDA approval for its drug Onpattro in August 2018. The culmination of a 16-year quest to realize the promise of Nobel-winning research, Onpattro treats peripheral nerve damage in people with hereditary amyloidosis, a rare and previously untreatable genetic mutation that causes abnormal proteins to build up in the body’s nerves and organs and interfere with tissue and organ function. RNAi treatments work further upstream than most existing medicines by selectively “silencing” the production of disease-causing proteins. Alnylam has five more RNAi-based drugs in late-stage trials, including for high cholesterol and acute hemophilia, with treatments for hepatitis and hereditary liver disease in early development. Recent studies suggest RNAi drugs could also target neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and ALS. Onpattro has a list price of $450,000, but the company refunds the full cost if results don’t match those obtained in trials.

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headquarters: Cambridge, MA
Alnylam Pharmaceuticals On The Web
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