Ionis Pharmaceuticals, Inc.

Ionis Pharmaceuticals, Inc. is a biotechnology company specializing in RNA-targeted medicines, headquartered in Carlsbad, California and founded in 1989. The company focuses on discovering and developing antisense oligonucleotide therapies and related RNA-directed technologies to treat serious and often rare diseases across neurology, cardiometabolic, and other high-need therapeutic areas. Ionis develops proprietary drug candidates on its antisense platform and uses a hybrid model: it commercializes certain rare-disease medicines directly or with co-promotion partners, while licensing other programs to larger pharmaceutical companies for broad late-stage development and global commercialization. Its marketed portfolio includes therapies for conditions such as spinal muscular atrophy, hereditary transthyretin-mediated amyloidosis, familial chylomicronemia syndrome, and SOD1-ALS, among others, reflecting its focus on targeted, mechanism-based treatments. Operating today as a fully integrated commercial-stage biotech with a deep clinical pipeline, Ionis plays a significant role in advancing RNA-based therapeutics within the global biopharmaceutical market.