Ionis Pharmaceuticals Inc (Ionis), formerly ISIS Pharmaceuticals Inc, focuses on the discovery and development of a new class of antisense drug candidates for the treatment of various diseases. The company operates its business through a single reportable business segment namely, Ionis Operations. It develops products based on its patented antisense technology.

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Ionis conducts research and development to advance its pipeline and pre-clinical pipeline candidates of antisense drugs for the treatment of various diseases. Its drug research focuses on diseases that are associated with RNA targets found in these tissues. The company expected to add eplontersen for the treatment of polyneuropathy; and tofersen used to treat superoxide dismutase 1 amyotrophic lateral sclerosis. Its major pipeline products include: Eplontersen for transthyretin amyloidosis (ATTR); Olezarsen for FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for hereditary angioedema (HAE); ION363 for the treatment of amyotrophic lateral sclerosis (ALS); Tofersen for treatment of SOD1-ALS; Pelacarsen for elevated lipoprotein(a), or Lp(a) and cardiovascular disease. Bepirovirsen for chronic hepatitis B virus, or HBV. In FY2022, the company spent US$833.1 million for research and development activities which is 141.8% of total revenue. The company owns various patents based on breach of its characteristic features such as chemically modified nucleosides and oligonucleotides, drug design motifs, ligand-conjugated antisense (lica) technology, therapeutic methods, oligonucleotide sequence and drug composition.