Ionis Pharmaceuticals, Inc.

Ionis Pharmaceuticals makes medicines that work by targeting RNA, which is the genetic instruction material in cells. The company sells several approved drugs for rare genetic diseases: SPINRAZA treats spinal muscular atrophy in children and adults, QALSODY treats ALS (a disease that causes muscle weakness), and WAINUA treats a nerve disease caused by abnormal protein buildup. They also have drugs for uncommon conditions affecting cholesterol and fat metabolism, and a growing pipeline of experimental treatments under development. The company partners with major pharmaceutical firms like Biogen...