Ionis Pharmaceuticals, Inc. (Ionis), formerly Isis Pharmaceuticals, is a commercial-stage biotechnology company founded in 1989 and headquartered in Carlsbad, California. The company pioneers RNA-targeted medicines, specializing in antisense oligonucleotides (ASOs) that inhibit disease-causing proteins by binding to specific RNA sequences.
Ionis has developed and commercialized several therapies for serious diseases, including SPINRAZA (nusinersen) for spinal muscular atrophy, TEGSEDI (inotersen) and QALSODY (tofersen) for hereditary transthyretin-mediated amyloidosis and ALS, respectively, WAYLIVRA (volanesorsen) for familial chylomicronemia syndrome, and WAINUA (eplontersen) for ATTRv-PN. Its pipeline targets neurology, cardiometabolic disorders, and rare diseases, with candidates like bepirovirsen (licensed to GSK for chronic hepatitis B) in late-stage development.
With over 1,400 employees, Ionis advances antisense technology alongside RNA interference and gene editing approaches through strategic partnerships with Biogen, GSK, AstraZeneca, Novartis, and Roche.
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Isis Pharmaceuticals.
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