Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals is dedicated to unlocking the potential of RNA- and DNA-targeted medicines to improve human health. With a history of pioneering science and technology, Ionis aims to develop innovative therapies for serious diseases across various focus areas including neurology, cardiology, and high unmet patient needs. The company emphasizes responsible operation, diversity, inclusion, and community support, striving to create a healthier and more resilient future for all.
Industries
Nr. of Employees
large (251-1000)
Ionis Pharmaceuticals, Inc.
Patents
Products
Pipeline of RNA- and DNA-targeted therapeutic candidates
A portfolio of investigational RNA- and DNA-targeted medicines in development across neurology, cardiology and select high-need areas (rare diseases, metabolic and renal indications).
Commercialized RNA-targeted therapy for a cardiometabolic indication
A marketed RNA-targeted medicine for a rare cardiometabolic disease that has undergone regulatory review and independent commercial launch preparations.
Pipeline of RNA- and DNA-targeted therapeutic candidates
A portfolio of investigational RNA- and DNA-targeted medicines in development across neurology, cardiology and select high-need areas (rare diseases, metabolic and renal indications).
Commercialized RNA-targeted therapy for a cardiometabolic indication
A marketed RNA-targeted medicine for a rare cardiometabolic disease that has undergone regulatory review and independent commercial launch preparations.
Services
A program to fund high-risk/high-reward academic projects to accelerate novel therapeutic technologies, including award administration and IP licensing terms.
Design and execution of clinical studies across multiple therapeutic areas and phases, including pivotal trials and data publication.
Electronic supplier portal onboarding, purchase order management, electronic invoicing (cXML/Actionable Email) and payment processing support for vendors.
Grant and sponsorship programs to support patient community initiatives, disease awareness, education and patient resources.
A program to fund high-risk/high-reward academic projects to accelerate novel therapeutic technologies, including award administration and IP licensing terms.
Design and execution of clinical studies across multiple therapeutic areas and phases, including pivotal trials and data publication.
Electronic supplier portal onboarding, purchase order management, electronic invoicing (cXML/Actionable Email) and payment processing support for vendors.
Grant and sponsorship programs to support patient community initiatives, disease awareness, education and patient resources.
Expertise Areas
- RNA and DNA therapeutics development
- Antisense oligonucleotide (ASO) design
- siRNA therapeutics
- Gene editing research
Key Technologies
- Antisense oligonucleotides (ASOs)
- siRNA
- Gene editing methods
- Modified oligonucleotide backbones (e.g., MsPA)
News & Updates
TRYNGOLZA TM delivers $19 million in net product sales in the second quarter 2025; Donidalorsen approval in hereditary angioedema (HAE) anticipated next month; Phase 3 data from the pivotal CORE and CORE2 studies in severe hypertriglyceridemia (sHTG)
Based on Phase 3 Balance results, which showed a significant reduction of triglycerides and substantial reduction of acute pancreatitis events with TRYNGOLZA. European Commission decision expected by Q4 2025.
Patients switched to donidalorsen without increased breakthrough attacks and experienced further reductions in mean attack rate from baseline. 84% of patients surveyed preferred donidalorsen over prior treatment. Currently under review with the U.S.
TRYNGOLZA TM delivers $19 million in net product sales in the second quarter 2025; Donidalorsen approval in hereditary angioedema (HAE) anticipated next month; Phase 3 data from the pivotal CORE and CORE2 studies in severe hypertriglyceridemia (sHTG)
Based on Phase 3 Balance results, which showed a significant reduction of triglycerides and substantial reduction of acute pancreatitis events with TRYNGOLZA. European Commission decision expected by Q4 2025.
Patients switched to donidalorsen without increased breakthrough attacks and experienced further reductions in mean attack rate from baseline. 84% of patients surveyed preferred donidalorsen over prior treatment. Currently under review with the U.S.