Ionis Pharmaceuticals
Biotechnology company focused on discovery, development and commercialization of RNA- and DNA-targeted medicines. Core competencies include discovery and optimization of oligonucleotide therapeutics (antisense and siRNA), oligonucleotide chemistry and conjugate-mediated delivery, preclinical translational science, clinical development and trial execution, GMP manufacturing and commercialization operations. Therapeutic focuses include neurology, cardiometabolic and select rare diseases.
Industries
Nr. of Employees
Very Large (1000+)
Ionis Pharmaceuticals
Carlsbad, California, United States, North America
Patents
Products
Approved oligonucleotide medicines (neurology, cardiometabolic, hereditary angioedema)
Commercial-stage RNA-targeted medicines approved for specific neurologic, cardiometabolic and hereditary angioedema indications with supporting prescribing information and post-approval programs.
Approved oligonucleotide medicines (neurology, cardiometabolic, hereditary angioedema)
Commercial-stage RNA-targeted medicines approved for specific neurologic, cardiometabolic and hereditary angioedema indications with supporting prescribing information and post-approval programs.
Services
End-to-end clinical development services including protocol design, clinical operations, investigational product supply management and data delivery.
Administration of grant and sponsorship programs to support independent clinical and translational research aligned with therapeutic priorities.
Milestone-driven awards for high-risk/high-reward laboratory projects to accelerate disruptive therapeutic technologies; includes defined IP terms and option/license provisions.
Services and infrastructure for product commercialization, including market access strategy, payer engagement and patient support services.
End-to-end clinical development services including protocol design, clinical operations, investigational product supply management and data delivery.
Administration of grant and sponsorship programs to support independent clinical and translational research aligned with therapeutic priorities.
Milestone-driven awards for high-risk/high-reward laboratory projects to accelerate disruptive therapeutic technologies; includes defined IP terms and option/license provisions.
Services and infrastructure for product commercialization, including market access strategy, payer engagement and patient support services.
Expertise Areas
- RNA-targeted therapeutics
- Antisense oligonucleotide therapeutics
- siRNA therapeutics and conjugate delivery
- Gene editing and epigenome modulation research
Key Technologies
- Antisense oligonucleotides (ASOs)
- Small interfering RNA (siRNA)
- Ligand- and peptide-conjugation delivery methods
- Modified oligonucleotide backbone chemistries
News & Updates
Regulatory approval of an RNA-targeted therapy as a disease-modifying treatment for Alexander disease in pediatric and adult patients.
Partner-announced topline results for a Phase 3 cardiovascular outcomes trial did not meet the primary endpoint.
Ranked #3 in the Large Company category in the San Diego Business Journal's Best Places to Work rankings.
Collection of peer-reviewed publications and presentations reporting clinical and translational data for cardiometabolic and other programs.
Public overview of the company clinical pipeline including approved medicines and investigational RNA-targeted programs across neurology, cardiometabolic and rare disease indications.
Patient stories and perspectives illustrating disease burden and patient needs for indications such as Alexander disease and severe hypertriglyceridemia.
Regulatory approval of an RNA-targeted therapy as a disease-modifying treatment for Alexander disease in pediatric and adult patients.
Partner-announced topline results for a Phase 3 cardiovascular outcomes trial did not meet the primary endpoint.
Ranked #3 in the Large Company category in the San Diego Business Journal's Best Places to Work rankings.
Collection of peer-reviewed publications and presentations reporting clinical and translational data for cardiometabolic and other programs.
Public overview of the company clinical pipeline including approved medicines and investigational RNA-targeted programs across neurology, cardiometabolic and rare disease indications.
Patient stories and perspectives illustrating disease burden and patient needs for indications such as Alexander disease and severe hypertriglyceridemia.