Oral PAR1-targeting drugs to reduce liver inflammation in MASH
Small Molecule PAR1 Ligands for the Treatment of MASH
This project develops new oral small-molecule drugs designed to shift liver PAR1 signaling and reduce inflammation in adults with MASH.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Function Therapeutics, INC. NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project ID | NIH-11163456 on NIH RePORTER |
What this research studies
The team is creating and testing small molecules called parmodulins that act on a receptor called PAR1 in the liver. They will synthesize oral-ready candidate compounds and test them in mouse models that mimic human MASH to see whether inflammation and downstream fibrosis are reduced. The approach aims to tip PAR1 signaling from a pro-inflammatory to an anti-inflammatory state, building on prior animal results with activated protein C and PAR1-modified mice. This is early-stage preclinical work at a biotech company and does not yet involve people, but successful compounds could move toward human trials later.
Who could benefit from this research
Good fit: Adults diagnosed with metabolic dysfunction-associated steatohepatitis (MASH), particularly those with obesity-related fatty liver, would be the likely candidates for future trials.
Not a fit: People with advanced cirrhosis, liver disease driven primarily by viruses or alcohol rather than metabolic dysfunction, or children are less likely to benefit from this early-stage program.
Why it matters
Potential benefit: If successful, these drugs could slow or reduce liver inflammation and fibrosis in people with MASH and lead to new oral treatment options.
How similar studies have performed: Preclinical mouse studies with activated protein C and PAR1-modifying compounds have shown reduced inflammation, but there are currently no approved PAR1-targeting drugs for MASH and human data are lacking.
Where this research is happening
Milwaukee, United States
- Function Therapeutics, INC. — Milwaukee, United States (Active)
Researchers
- Principal investigator: Dockendorff, Christopher — Function Therapeutics, INC.
- Study coordinator: Dockendorff, Christopher
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.