Ion Holdings

Ionis Pharmaceuticals is a leading biotechnology company specializing in the discovery and development of RNA-targeted therapies, particularly antisense oligonucleotides (ASOs). Founded in 1989 as Isis Pharmaceuticals, the company pioneered the use of synthetic nucleic acids to selectively bind to messenger RNA and modulate the production of disease-causing proteins. Ionis has successfully brought several first-in-class medicines to market, including Spinraza for spinal muscular atrophy and Tegsedi for hereditary transthyretin amyloidosis. Its robust pipeline spans neurology, cardiometabolic diseases, and rare genetic disorders, utilizing advanced chemistries like Ligand-Conjugated Antisense (LICA) to enhance potency and delivery. The company operates through strategic partnerships with major pharmaceutical firms while increasingly focusing on independent commercialization of its wholly owned programs.