Ionis Pharmaceuticals, Inc.

Ionis Pharmaceuticals, Inc. is a biotechnology company headquartered in Carlsbad, California, dedicated to discovering and developing RNA-targeted therapeutics. The company's mission is to deliver life-transforming therapies to people with serious diseases by pioneering RNA-targeted therapeutics, aiming to translate the promise of RNA-based medicines into life-changing treatments for patients.

Ionis specializes in antisense therapy, RNA interference, and CRISPR therapeutics, focusing on a broad range of diseases including neurological, cardiometabolic, and rare genetic disorders. Key marketed products include Spinraza (nusinersen) for spinal muscular atrophy, Tegsedi (inotersen) and Wainua (eplontersen) for hereditary transthyretin-mediated amyloidosis, Waylivra (volanesorsen) for familial chylomicronemia syndrome, Qalsody (tofersen) for a genetic form of ALS, TRYNGOLZA (olezarsen) for familial chylomicronemia syndrome, and DAWNZERA (donidalorsen) for hereditary angioedema.

In recent news, Ionis Pharmaceuticals reported strong first-quarter 2026 financial results, surpassing Wall Street expectations and leading to an increase in its full-year 2026 revenue guidance. The company's independent product launches, particularly TRYNGOLZA and DAWNZERA, have significantly contributed to revenue growth. Ionis continues to advance its pipeline, with the U.S. FDA accepting New Drug Applications for donidalorsen for prophylactic treatment of HAE, olezarsen for Priority Review in severe hypertriglyceridemia, and zilganersen for Alexander disease, positioning the company for further independent launches. Brett P. Monia serves as the Chief Executive Officer.