Ionis Pharmaceuticals

Ionis Pharmaceuticals

IONSApproved
Carlsbad, United StatesFounded 1989ionis.com

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.

Market Cap
$11.7B
Founded
1989
Focus
Drug DeliveryRNA & Gene TherapySmall Molecules

IONS · Stock Price

USD 70.99+34.32 (+93.59%)

Historical price data

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

134

134 drugs in pipeline, 29 in Phase 3

DrugIndicationStage
ION582 + PlaceboAngelman SyndromePhase 3
Inotersen + EplontersenHereditary Transthyretin-Mediated Amyloid PolyneuropathyPhase 3
OlezarsenFamilial Chylomicronemia SyndromePhase 3
ISIS 3521 + carboplatin + paclitaxelLung CancerPhase 3
mipomersen + PlaceboLipid Metabolism, Inborn ErrorsPhase 3

Funding History

3

Total raised: $133M

PIPE$100MRoyalty PharmaDec 15, 2015
Series A$5MUndisclosedJun 15, 1991
IPO$28MUndisclosedMay 15, 1991

FDA Approved Drugs

2
DAWNZERA (AUTOINJECTOR)NDAAug 21, 2025
TRYNGOLZA (AUTOINJECTOR)NDADec 19, 2024

Opportunities

The company is well-positioned to capture increased value from its proprietary medicines as a fully integrated biotechnology company, with multiple late-stage programs approaching commercialization and a strong foundation in RNA therapeutics providing opportunities across numerous genetic diseases.

Risk Factors

Key risks include clinical trial and regulatory uncertainties, execution challenges in building commercial capabilities as a newly integrated company, and increasing competition in the rapidly evolving RNA therapeutics space.

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.

Publications
19
Patents
20
Pipeline
134
FDA Approvals
2

Company Info

TypeTherapeutics
Founded1989
LocationCarlsbad, United States
StageApproved
RevenueRevenue Generating

Trading

TickerIONS
ExchangeNASDAQ

Contact

Therapeutic Areas

NeurologyCardiologyRare DiseasesMetabolic DisordersGenetic Diseases

Partners

Multiple biotechnology and pharmaceutical companies for medicine commercializationOver 100 patient advocacy organizations
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