Experimental Drug That Mutes Defective Genes Raises New Hopes

October 5, 2017

(Scientific American) – The RNAi delivery systems remain highly complex—and the most effective technologies are still protected by patents that make it difficult for startups to get into the field. Safety concerns persist with other RNAi drugs in development: Last year, for instance, Alnylam had to scrap revusiran, one of its most advanced drugs. Rather than alleviating it, the drug exacerbated pain in a rare nerve disease called transthyretin amyloidosis. And several patients died in the clinical trial, though it’s still not clear exactly why. Alnylam’s stock plummeted by half on that news.

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