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A Triumph of Perseverance Over Interference [Nature Biotechnology]
September 10, 2018
Alnylam Pharmaceuticals
The following Editorial appeared in Nature Biotechnology (36,775 2018) on September 6, 2018...
"A triumph of perseverance over interference
The US approval of Onpattro—the first of a new class of small-interfering RNA (siRNA) drug—is a triumph for Alnylam and vindication for its leadership.
On August 10, the US Food and Drug Administration approved Onpattro (patisiran), Alynlam Pharmaceuticals' trailblazing siRNA treatment for neuropathy in hereditary transthyretin amyloidosis (hATTR). The approval validates siRNA as a new type of disease-modifying treatment that coopts the cell's endogenous microRNA (miRNA) machinery to downregulate aberrant mRNAs. It also represents the culmination of a $2.5 billion drug development program that took just 16 years—a remarkably short time to bring an entirely new therapeutic class to market, especially when one considers the challenges in terms of delivery, immunogenicity, specificity and stability. That Onpattro made it over the finish line is testament to the experience and perseverance of Alnylam's leadership. But for RNA interference (RNAi) to establish its credentials as a bona fide treatment modality, more siRNA therapies must follow in the near future."
Click here to read the full article on Nature Biotechnology website.
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