Ionis Pharmaceuticals Inc

Ionis Pharmaceuticals, Inc. is engaged in discovering and developing ribonucleic acid (RNA)-targeted therapeutics. The Company is primarily focused on two core franchises: neurology and cardiometabolic. Its products include SPINRAZA, TEGSEDI and WAYLIVRA. SPINRAZA is for treatment of patients of all ages 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. Its products in pipeline include tofersen for SOD1-ALS, eplontersen (IONIS-TTR-LRX) for TTR, IONIS-APOCIII-LRx for familial chylomicronemia syndrome (FCS), pelacarsen for lipoprotein(a) (Lp), driven cardiovascular disease and ION363 for amyotrophic lateral sclerosis (ALS), with mutations in the fused in sarcoma gene (FUS).