Areas of Focus

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 cardiovascular disease

Cardiovascular disease (CVD) is the leading cause of death globally. Given the vast numbers of people impacted, we are developing cardiovascular medicines that target major conditions that cause heart and metabolic disease. Our pipeline includes investigational medicines for familial chylomicronemia syndrome (FCS), severe hypertriglyceridemia (sHTG), transthyretin amyloid cardiomyopathy (ATTR-CM), and lipoprotein(a) [Lp(a)]-driven CVD.

Advancing medicines for rare diseases and other areas of patient need

Many rare 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 rare diseases 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.