Ionis Has Been at Work for Three Decades Discovering Medical Breakthroughs That Have Redefined Life for People With Serious Diseases, and We Are Confident That Our Most Important Breakthroughs Lie Ahead
As we look to the future, we are building on our rich history in pioneering RNA-targeted medicines to continue transforming outcomes for patients in our focus areas of neurology, cardiometabolic disease and select areas of high patient needs.
Addressing a broad range of rare and common neurologic diseases
There is a significant need for disease modifying treatments for people living with severe neurologic diseases. We’ve been successful in delivering first-in-class medicines for diseases that were previously untreatable, and we’re determined to bring the same hope to patients with other neurologic conditions where the needs are profound. We’re investigating novel medicines for rare neurologic diseases, including Alexander disease, Angelman syndrome, prion diseases, Pelizaeus-Merzbacher disease, MECP2 duplication syndrome, and amyotrophic lateral sclerosis, and more prevalent neurologic diseases, including Alzheimer’s disease and Parkinson’s disease.
Targeting major risk factors for cardiometabolic disease
Cardiometabolic disease is among the most pressing health challenges of our time. Given the vast numbers of people impacted, we are developing medicines that target major conditions that cause heart and metabolic disease. We delivered an approved medicine for familial chylomicronemia syndrome (FCS) and our pipeline includes investigational medicines for severe hypertriglyceridemia (sHTG), transthyretin amyloid cardiomyopathy (ATTR-CM), and lipoprotein(a) [Lp(a)]-driven CVD.
Advancing medicines for select areas of high patient needs
Many diseases have limited or no treatment options, so delivering on innovative science that will lead to new treatments is crucial. Because we recognize that life-changing medicines are needed for other areas outside of cardiology and neurology, we’re currently advancing the clinical development of investigational medicines for hereditary angioedema (HAE) and polycythemia vera. Beyond rare diseases, we’re also driving therapeutic development in additional areas – from renal and metabolic diseases to infectious diseases – where our science can provide new solutions for patients.
View Our Clinical Pipeline
We are relentlessly pursuing the innovations of tomorrow with our broad and diverse pipeline of potential first or best-in-class investigational therapies designed to treat a range of diseases. Learn more about our pipeline.