Treating Chronic Diseases Associated with Mitochondrial Pathophysiology with a Novel siRNA Approach
Time: 11:00 am
day: Day Two
Details:
- Demonstrated the role of MCJ, a key mitochondrial regulator, in NASH, related liver diseases, kidney and cardiovascular disease
- Developed a liver-targeted siRNA as a therapeutic approach to treating NASH; efficacy demonstrated in multiple preclinical disease models
- Conducting safety pharmacology and toxicity studies to support an IND filing with no adverse findings thus far
