Ionis scores ‘breakthrough’ rare disease approval
Ionis scores ‘breakthrough’ rare disease approval
Ionis Pharmaceuticals
Ionis Pharmaceuticals (NASDAQ: IONS) is a Carlsbad, California-based antisense oligonucleotide (ASO) drug discovery and development company with a deep pipeline spanning neurology, cardiometabolic disease, renal disease, and rare disorders.
Company Info
Sector:Antisense RNA Therapeutics
Headquarters:Carlsbad, California, USA
Founded:1989
Website:ionis.com/
Upcoming Milestones & Key Dates
2026-08-05
Earnings
Q2 2026 earnings
2026-10-15
Conference
EHA / ASH presentations
Clinical Development Pipeline
Tryngolza (olezarsen)
Target: APOC3 mRNAWainua (eplontersen)
Target: TTR mRNASapablursen
Target: TMPRSS6 mRNARecent News
Ionis wins FDA clearance for Alexander disease drug, company's first solo neurology approval
Ionis Pharmaceuticals receives FDA approval for its Alexander disease drug zilganersen, marking the company's first solo neurology approval.
FDA approves Ionis’ antisense drug as first targeted therapy for Alexander disease
Article from BioSpace about FDA approves Ionis’ antisense drug as first targeted therapy for Alexander disease.
FDA Approves Ionis Pharma Drug, the First for Ultra-Rare Alexander Disease
FDA Approves Ionis Pharma Drug, the First for Ultra-Rare Alexander Disease
AstraZeneca, Ionis’ Wainua woes send waves across ATTR-CM space
BioSpace reports: AstraZeneca, Ionis’ Wainua woes send waves across ATTR-CM space.
Roche scraps 2 Huntington’s studies, handing Ionis more bad news
BioSpace reports: Roche scraps 2 Huntington’s studies, handing Ionis more bad news.
Ionis secures market expansion for Tryngolza as FDA clears severe hypertriglyceridemia use
Ionis Pharmaceuticals secured an additional FDA approval for Tryngolza (olezarsen), expanding its market to include severe hypertriglyceridemia beyond the original familial chylomicronemia syndrome (FCS) indication.
Leadership Team
Brett P. Monia
Chief Executive Officer
Elizabeth L. Hougen
Chief Financial Officer
C. Frank Bennett
Chief Scientific Officer