Science That
Changes Lives
Where Science Meets Life
Scientific innovation is just the beginning of our mission. We push the boundaries of science to improve lives of patients and their loved ones, discovering new paths to a healthier future.
Cardiometabolic
Cardiometabolic diseases are a group of conditions with interrelated risk factors, primarily hypertension, elevated blood sugar, dyslipidemia, abdominal obesity, and elevated triglycerides. We’re committed to addressing the unmet needs in this area, across a spectrum of diseases.
Pulmonary
For people with pulmonary diseases, breath isn’t taken for granted. We’re dedicated to developing new therapies targeted at underlying causes including inflammation and over-production of mucus across a broad range of conditions impacting the lungs, such as chronic obstructive pulmonary disease, idiopathic pulmonary fibrosis, and more.
Liver
Some liver diseases, such as Alpha1- liver disease and non-alcoholic steatohepatitis (NASH), have limited options. Through dedicated research, we’re discovering and developing novel approaches for these intractable diseases.
Neuromuscular
Neuromuscular diseases are often progressive, debilitating, and could contribute to premature death. We’re pioneering new therapies with the goal of altering the course of neuromuscular diseases and meaningfully improving lives.
Other Areas
We see endless possibilities in our pursuit of novel approaches to treating disease. Every day we strive to identify and develop therapeutic targets that will have significant impact and improve outcomes.
Our Targeted RNAi Molecule: TRiM™
The TRiM™ platform is designed to overcome barriers associated with gene-silencing treatments, including targeting delivery of the RNAi drug to specific cells in the body for a sustained duration of activity, and preventing the drug from affecting off-target genes.
What is RNAi?
Many diseases are caused by excessive or abnormal production of a specific protein, by a specific gene.
RNAi, or RNA interference, is a natural cellular process that inhibits gene expression and regulates protein production. This process opens many opportunities in drug development, including the ability to target a wide range of genes and proteins with high specificity.
Because RNAi is a normal biological process, expression of any gene can be potentially “silenced” using RNAi.
Areas of Focus
Discover our areas of expertise and how we’re working to address unmet clinical needs.
Clinical Trials
Our clinical trials are an essential part of our research. Discover the therapies we’re currently developing.