Alnylam Pharmaceuticals is a relatively small Boston-based company, founded in 2002, that has been burning through cash – last year, it spent more than $0.5 billion and made only about $100 million. Its lead product, revusiran, failed in clinical trials in 2016, but Alnylam has nonetheless kept up some momentum, including interest from pharmaceutical giant Sanofi (the company bought a minority share of Alnylam), as well as an acquisition of one of Merck's subsidiaries, Sirna Therapeutics. In mid-2018, Alnylam's efforts were rewarded by the U.S. FDA, with Alnylam's Onpattro drug receiving the first-ever RNA interference drug approval. Onpattro is aimed at patients suffering from hereditary transthyretin-mediated amyloidosis (hATTR), a rare neurodegenerative brain disease affecting about 50,000 people worldwide.
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