Key information

🔎 Fund Overview

Ionis Pharmaceuticals, Inc. is engaged in discovering and developing ribonucleic acid (RNA)-targeted therapeutics. The Company is primarily focused on cardiovascular and neurology franchises. The Company’s products include SPINRAZA, TEGSEDI and WAYLIVRA. SPINRAZA is for the treatment of patients with spinal muscular atrophy (SMA), a progressive, debilitating and often fatal genetic disease. TEGSEDI is for the treatment of patients with polyneuropathy caused by hereditary transthyretin (TTR), amyloidosis (hATTR), a debilitating, progressive, and fatal disease. WAYLIVRA is an antisense medicine indicated as an adjunct to diet in adult patients. The Company has over seven medicines in Phase III studies for nine indications, which include Eplontersen (TTR), Olezarsen (ApoC-III), Donidalorsen (PKK), ION363 (FUS), Pelacarsen (Apo(a)), Tofersen (SOD1) and Bepirovirsen. Its cardiovascular medicine in development includes Eplontersen, Olezarsen, Pelacarsen, Fesomersen, ION904 and IONIS-AGT-LRx.