IONIS PHARMACEUTICALS INC

Ionis Pharmaceuticals is a commercial-stage biotechnology company developing RNA-targeted medicines for rare and specialty diseases. The company's marketed products include SPINRAZA for spinal muscular atrophy, QALSODY for amyotrophic lateral sclerosis, WAINUA and TEGSEDI for hereditary transthyretin amyloidosis, and TRYNGOLZA, WAYLIVRA for familial chylomicronemia syndrome and related lipid disorders. These therapies work by modulating RNA to address underlying genetic drivers of disease.

The company maintains a substantial clinical pipeline with multiple programs in Phase 3 development, including Olezarsen for hypertriglyceridemia and cardiovascular disease, Donidalorsen for hereditary angioedema, and Eplontersen for transthyretin amyloidosis. Additional mid-stage candidates target conditions including Alexander disease, hepatitis B, and other rare genetic disorders. Revenue is generated through product sales from approved medicines and milestone payments from strategic partnerships.

Ionis operates through collaboration agreements with major pharmaceutical companies including Biogen, GSK, AstraZeneca, Novartis, and Roche, which provide both funding and commercialization support. The company is headquartered in Carlsbad, California and maintains operations across the United States. Founded in 1989, Ionis focuses on a targeted therapeutic modality rather than broad disease categories, concentrating on markets where RNA-targeting approaches offer distinct clinical advantages.