A range of RNAi therapies are moving through clinical development and toward the market providing the promise of new ways to treat genetic diseases. Dicerna, which has a platform that allows for a unique delivery mechanism for targeting the liver with RNAi therapies, recently began dosing patients in an early-stage trial of its experimental RNAi therapy to treat hyperoxaluria, a rare genetic liver disease. We spoke to Doug Fambrough, president and CEO of Dicerna, about the company’s GalXC platform, what makes it unique, and what’s ahead in its pipeline.
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