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siMCJ
- Development stage
- Preclinical
- Lead developer
- Mitotherapeutix
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous, Subcutaneous
01
Overview
siMCJ is a small interfering RNA (siRNA) therapeutic designed to target and silence the expression of Methylation-Controlled J Protein (MCJ, also known as DNAJC15). MCJ is an endogenous negative regulator of the mitochondrial electron transport chain (ETC) Complex I, acting as a metabolic brake. By silencing MCJ, siMCJ enhances Complex I activity, mitochondrial respiration, and ATP production without increasing reactive oxygen species (ROS). Developed by Mitotherapeutix in collaboration with researchers at the University of Colorado Anschutz Medical Campus, siMCJ is being investigated for the treatment of metabolic, fibrotic, and inflammatory diseases, including non-alcoholic steatohepatitis (NASH), acute kidney injury (AKI), autosomal dominant polycystic kidney disease (ADPKD), and acetaminophen (APAP)-induced liver injury.
- Other names
- siRNA-based MCJsiRNA targeting MCJ
02
Targets
DNAJC15 (DnaJ heat shock protein family member C15)
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