A new siRNA drug to treat fatty liver disease by boosting liver energy production
Official title: 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)
Funded at Mitotherapeutix, LLC · NIH-11312337 · Data updated 2026-11-12
A new siRNA drug aimed at reducing liver fat, inflammation, and fibrosis in people with metabolic dysfunction-associated steatohepatitis (MASH).
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mitotherapeutix, LLC NIH-funded |
| Lab location | 1 site (Cheshire, United States) |
| Project start | June 1, 2021 |
| Project end | July 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $996,169 |
| NIH project number | 2R44DK129102-04A1 |
| Project ID | NIH-11312337 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with metabolic dysfunction-associated steatohepatitis (MASH) who are candidates for early-phase clinical testing.
✕Less likely to help
Patients without MASH or those not eligible for early-phase trials may not benefit.
What this research studies
This project focuses on making a novel siRNA drug that works in the liver to increase mitochondrial metabolism, helping the liver burn fatty acids more effectively. The plan includes cGMP production, IND-enabling studies, and early Phase I clinical testing to see if the treatment is safe and feasible in people with MASH. If successful, it could lead to better control of liver fat and scarring, potentially slowing progression to cirrhosis or liver failure. The work is led by MITOTHERAPEUTIX, LLC, with a physician-scientist team exploring how targeting MCJ could improve liver health.
Why it matters
Potential benefit: If successful, patients with MASH could experience reduced liver fat and inflammation, potentially slowing disease progression.
How similar studies have gone: This approach builds on prior understanding of mitochondrial metabolism in liver disease, with early signals suggesting safety and feasibility in related models.
Where this research is happening
-
Mitotherapeutix, LLC
Cheshire, United States Active
Researchers
- Principal investigator: Arbeeny, Cynthia — Mitotherapeutix, LLC
- Study coordinator: Arbeeny, Cynthia
Related research
Other NIH-funded projects on the same topics.
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Regulation of ROBO1 by YAP and LHX2 in MASLD-related liver disease
A study at Tulane is exploring how certain cell signals influence liver scarring and regeneration in fatty liver disease to find ways to reduce fibrosis.
-
Aldehydes and fat-related liver disease: how tiny molecules drive MASH in diabetes risk
Aims to understand how aldehydes created in fatty liver may trigger liver damage and inflammation in people with obesity and type 2 diabetes.
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Endohepatology-based reclassification of treatment indication in high-risk MASLD
This study tests whether an endohepatology assessment can change treatment decisions for adults with high-risk MASLD.
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Pre-hepatectomy rehabilitation with mazdutide for obese MASLD donors
This trial tests whether adding mazdutide to a structured lifestyle plan helps overweight MASLD donors lose liver fat faster to become eligible for donation.
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Efimosfermin vs. placebo in adults with compensated cirrhosis from MASH
This study tests whether Efimosfermin can help adults with compensated cirrhosis due to MASH compared with placebo.
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Efimosfermin vs placebo in adults with compensated cirrhosis from MASH
This study tests whether efimosfermin helps adults with compensated cirrhosis caused by MASH compared with placebo.
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.