Ionis Pharmaceuticals, Inc.
Ionis (formerly Isis) Pharmaceuticals is trying to make sense out of antisense. The biotech develops drugs based on its antisense technology, in which drugs attach themselves to strands of RNA in order to prevent them from producing disease-causing proteins; the hoped-for end result is a therapy that fights disease without harming healthy cells. Ionis has nearly 40 pipeline drugs under development in areas including cardiovascular, metabolic, and severe and rare diseases (including cancer and neurological disorders). Its lead product is the cholesterol-lowering drug Kynamro, which Ionis has licensed to Genzyme; it is marketed in the US and other countries for the treatment of genetic disorder homzygous FH.
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