Our lead clinical-stage candidates are focused on treating a wide range of diseases including metabolic, muscle and neurological diseases, including:
Nonalcoholic fatty liver disease (NAFLD) is a disease where excessive fat accumulates in the liver of a patient with no history of alcohol abuse. Non-alcoholic steatohepatitis (NASH) is a more severe form of NAFLD with liver inflammation and fibrosis, putting patients at greater risk of developing cirrhosis. These patients may ultimately require liver transplants.
Muscle loss in aging, during hospitalization, or from neuromuscular diseases are major areas of unmet need. Skeletal muscle loss (atrophy) occurs when muscle protein breakdown exceeds synthesis. Progressive muscle loss correlates with co-morbidities including physical disability, increased likelihood of falls, poor quality of life and death.
Intractable (Drug-Resistant) Epilepsy Syndromes
Excessive and abnormal brain cell activity leads to seizures that can greatly disrupt a person’s life, causing physical injury and even death. About one-third of people with epilepsy are being treated with medicines that do not work well, or at all, to control their seizures.
New Disease Opportunities
In addition we have a rich, therapeutically diverse pipeline of newly identified disease opportunities that are rapidly advancing through development.