Cynthia Arbeeny
Seminars
Monday 29th September 2025
10.15 am MITO-1041: A Unique Liver-Directed siRNA Approach to Treat Mitochondrial Dysregulation in MASH
- Mitochondrial dysregulation is a key driver for the initiation and progression of MASH
- MCJ is a key regulator of mitochondrial metabolism and knockdown improves steatosis, inflammation and fibrosis in MASH preclinical models
- MITO-1041 is a GalNAc MCJ siRNA which shows promise as a safe and effective therapy that can be used alone or in combination for treating patients with MASH
