Ionis Pharmaceuticals, Inc. is engaged in ribonucleic acid (RNA)-targeted therapeutics. The Company has five marketed medicines and a pipeline in neurology, cardiology, and other areas of high patient need. It has nine medicines in Phase III development and multiple additional medicines in early and mid-stage development. The Companyâs products include SPINRAZA, QALSODY, WAINUA, TEGSEDI and WAYLIVRA. SPINRAZA is used for the treatment of patients with spinal muscular atrophy (SMA), a progressive, debilitating and often fatal genetic disease. QALSODY is an antisense medicine that is used for the treatment of adult patients with superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS), a rare, neurodegenerative disorder that causes progressive loss of motor neurons leading to death. WAINUA is a self-administered subcutaneous LIgand-Conjugated Antisense (LICA) medicine used for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN).
IONIS PHARMACEUTICALS, INC. 
