Ionis Pharmaceuticals
IONSApprovedFounded in 1989 by Stanley T. Crooke, Ionis Pharmaceuticals is a pioneer in RNA therapeutics with over three decades of innovation in antisense oligonucleotide technology. The company has successfully transitioned to a fully integrated biotechnology organization, launching its first independent medicine TRYNGOLZA (olezarsen) for familial chylomicronemia syndrome. With six currently marketed medicines discovered and developed by Ionis, the company maintains a rich late-stage pipeline focused primarily on neurology and cardiology, while continuing to advance breakthrough RNA-targeted therapies that have redefined treatment for serious genetic diseases.
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AI Company Overview
Founded in 1989 by Stanley T. Crooke, Ionis Pharmaceuticals is a pioneer in RNA therapeutics with over three decades of innovation in antisense oligonucleotide technology. The company has successfully transitioned to a fully integrated biotechnology organization, launching its first independent medicine TRYNGOLZA (olezarsen) for familial chylomicronemia syndrome. With six currently marketed medicines discovered and developed by Ionis, the company maintains a rich late-stage pipeline focused primarily on neurology and cardiology, while continuing to advance breakthrough RNA-targeted therapies that have redefined treatment for serious genetic diseases.
Technology Platform
Proprietary antisense oligonucleotide platform that targets RNA to modulate protein production, utilizing advanced chemistry including phosphorothioate substitutions and 2′-O-methoxyethyl modifications for enhanced stability and target affinity.
Pipeline Snapshot
134134 drugs in pipeline, 29 in Phase 3
| Drug | Indication | Stage |
|---|---|---|
| ION582 + Placebo | Angelman Syndrome | Phase 3 |
| Inotersen + Eplontersen | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Phase 3 |
| Olezarsen | Familial Chylomicronemia Syndrome | Phase 3 |
| ISIS 3521 + carboplatin + paclitaxel | Lung Cancer | Phase 3 |
| mipomersen + Placebo | Lipid Metabolism, Inborn Errors | Phase 3 |
Funding History
3Total raised: $133M
FDA Approved Drugs
2Opportunities
Risk Factors
Competitive Landscape
Ionis competes in the RNA therapeutics market with companies developing various nucleic acid-based approaches, but maintains differentiation through its pioneering position in antisense technology, extensive clinical experience with six marketed medicines, and proprietary chemistry platforms refined over three decades.
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