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Biotech leaders call for free press

To the Editor: We, the undersigned, are biotechnology executives, entrepreneurs, academic leaders and investors. We are gravely concerned about trends in the United States that are undermining our news media, such that more than 300 news publications across the country recently found it necessary to run coordinated editorials in defense of the First Amendment’s guarantee of freedom of the press.  Read more

Alnylam launches era of RNAi drugs

Alnylam launches era of RNAi drugs

On August 10, the US Food and Drug Administration approved the first RNA interference (RNAi) therapeutic, a treatment for polyneuropathy caused by transthyretin (TTR) amyloidosis from Alnylam Therapeutics. The go-ahead for Onpattro (patisiran) sees the RNAi field clear an approval hurdle considered unlikely as recently as six years ago, when pharma exited the RNAi field en masse. The US approval, with Europe expected to follow by early September, is “a major milestone,” says Anastasia Khvorova, an RNAi researcher at the University of Massachusetts in Worcester. Onpattro has an excellent safety record, but there are lingering concerns about potential long-term toxicity from newer, more potent RNAi therapeutics. And the field as a whole still faces investor skepticism in the wake of a decade of clinical trial failures.  Read more