US regulators have approved the first therapy based on RNA interference (RNAi), a technique that can be used to silence specific genes linked to disease. The drug, “patisiran“, targets a rare condition that can impair heart and nerve function. The approval is a landmark for a field that has struggled for nearly two decades to prove its worth in the clinic. Researchers first discovered RNAi 20 years ago, sparking hopes of a revolutionary new approach to medicine. Since then, however, a series of setbacks has lessened those expectations.

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