Ionis Pharmaceuticals, Inc.
A biotech pioneer that designs medicines that target RNA—the body's genetic messengers—to treat diseases that were long considered "undruggable." Its medicines include SPINRAZA for spinal muscular atrophy and newer launches like WAINUA, TRYNGOLZA, and DAWNZERA, used mainly by patients with rare, neurological, and cardiometabolic conditions. Founded in 1989, it was originally named Isis Pharmaceuticals after the Egyptian goddess of healing, but renamed itself Ionis in 2015 to shake an unfortunate association with the militant group of the same name.