Ionis Pharmaceuticals, Inc.

Ionis Pharmaceuticals, Inc. is a biotechnology company specializing in the discovery, development, and commercialization of human therapeutics using antisense technology. This proprietary platform enables the creation of novel drugs targeting difficult-to-treat diseases across cardiovascular, metabolic, neurological, and rare disease areas. Key marketed products include TRYNGOLZA (olezarsen), the first FDA-approved treatment for adults with familial chylomicronemia syndrome as an adjunct to diet; DAWNZERA (donidalorsen), an RNA-targeted prophylactic therapy for hereditary angioedema in patients 12 years and older; and royalties from partnered drugs such as SPINRAZA (nusinersen) for spinal muscular atrophy, WAINUA (eplontersen) for polyneuropathy of hereditary transthyretin-mediated amyloidosis, Qalsody for ALS, Tegsedi, and Waylivra. Ionis maintains a broad pipeline of investigational medicines, including olezarsen for severely elevated triglycerides, zilganersen for Alexander disease, bepirovirsen for chronic hepatitis B, ulefnersen for FUS-ALS, sapablursen for polycythemia vera, and opemalirsen for APOL1-mediated kidney disease. The company generates revenues from product sales, royalties, and R&D collaborations with partners like Biogen and AstraZeneca. Founded in 1989 and headquartered in Carlsbad, California, Ionis Pharmaceuticals plays a pivotal role in advancing RNA-targeted therapies for unmet medical needs.