Award
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.