LysoGen: a targeted liver therapy to boost lysosome health in MASLD
Official title: Lysosomal Regeneration (LysoGen) and Rescue as a Treatment for Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)
Funded at Boston University (Charles River Campus) · NIH-11382288 · Data updated 2026-11-12
A new medicine idea aims to restore normal lysosome acidity in the liver to help people with MASLD.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston University (Charles River Campus) NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 20, 2024 |
| Project end | May 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $619,776 |
| NIH project number | 5R01DK141923-03 |
| Project ID | NIH-11382288 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are adults with MASLD or metabolic dysfunction-associated fatty liver disease who could be eligible for future translational testing of liver-directed lysosomal therapies.
✕Less likely to help
Patients without MASLD or with advanced liver disease not suitable for this type of intervention may not benefit.
What this research studies
This project proposes an acid-activated therapy that travels to the liver and releases acid inside lysosomes to re-balance their pH and restore autophagy. By improving lysosome function, it seeks to reduce buildup of fats and damaged cell components that drive MASLD. The investigators will test a polyester-based material that responds to the lysosome’s acidity after intravenous delivery. If successful, this approach could complement or replace current liver-directed treatments that don’t target lysosomes directly. The work is early-stage and will focus on proving the concept and safety in preclinical models.
Why it matters
Potential benefit: If successful, the therapy could improve liver function and slow MASLD progression by reactivating the cell’s waste-disposal system.
How similar studies have gone: Previous work shows that targeting lysosome function can influence liver cell health, but this exact acid-activated LysoGen approach is novel.
Where this research is happening
-
Boston University (Charles River Campus)
Boston, United States Active
Researchers
- Principal investigator: Grinstaff, Mark W. — Boston University (Charles River Campus)
- Study coordinator: Grinstaff, Mark W.
Related research
Other NIH-funded projects on the same topics.
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Them1’s role in fat metabolism and MASLD progression
Aims to understand how a fat-regulating protein (Them1) affects energy use in fat and liver, and how this could contribute to MASLD in people who are overweight.
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Understanding MASLD progression to MASH and liver cancer in Alaska Native communities
A patient-facing overview of a study that looks at why a common liver disease progresses to more serious conditions in Alaska Native and American Indian people.
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Pediatric MASLD: a blood test to detect liver disease progression in kids
A child-friendly blood test is being developed to spot liver disease progression in kids with MASLD without needing biopsies.
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Growth Hormone and DNA Repair in Children's MASLD outcomes
A small, patient-friendly study will explore how growth hormone and the ATM DNA repair pathway affect liver health in children with MASLD.
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Isocaloric navy bean substitution for MASLD with fibrosis
A small, patient-friendly trial tests whether swapping in navy beans for some daily calories can help adults with MASLD and liver fibrosis without changing total calories.
-
S100A9+ immune cells driving liver scarring in MASLD/MASH
Affects people with MASLD/MASH may benefit from a better understanding of how certain immune cells contribute to liver fibrosis and how to stop it.
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.