# NIH Reporter #2R44DK129102-04A1

cGMP Production, IND-enabling Studies, and Phase I Clinical Studies to Test a Novel siRNA Drug Product for the Treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH)

**Recipient:** MITOTHERAPEUTIX, LLC

**Award Amount:** $996,169.00
**Ceiling:** $996,169.00

**Awarded:** September 17, 2026

**Identifier:** 2R44DK129102-04A1

The NIH-funded project supports development of MITO-1041, a novel siRNA therapy intended to treat MASH by silencing MCJ and increasing mitochondrial metabolism in the liver.

### Description

Funding to develop MITO-1041, an siRNA drug candidate targeting MCJ for treatment of MASH. The project includes cGMP manufacturing, assay development and validation, IND-enabling toxicology and other studies, submission of an IND, and a Phase I clinical study.

[View original record](https://reporter.nih.gov/search/J7l2tQ4hHkqRePwGq3m5kA/project-details/11312337)