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European Biopharmaceutical Review

GOAL!

Alnylam’s Onpattro is the first approved drug that treats disease by RNA interference – silencing gene expression. It is the culmination of a 15-year-long march by the company to develop and harness the enabling technologies necessary for successful commercialisation. Some commentators have called it a “clean sweep of efficacy and safety.”

The FDA views this breakthrough as part of a growing trend to treat disease by targeting root cause instead of treating symptoms. In this regard, RNA interference is complementary to other advanced therapeutic approaches. RNAi, like monoclonal antibodies, is dosedependent and can be modulated. Gene therapy, when it comes to fruition, is, in principle, permanent.

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Emile Bellott is a member of the EBR Industry Advisory Board, a graduate of the Harvard Business School, US, and an industry consultant with experience in the biotech and biopharma industry.
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Emile Bellott
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