Small-molecule drug discovery for heart damage in Duchenne muscular dystrophy using AI and patient-derived heart cells
Official title: Small Molecule Drug Discovery for Cardiac Fibrosis using AI/ML and iPSC-Derived Organoids
Funded at Greenstone Biosciences, INC. · NIH-11282472 · Data updated 2026-11-12
Researchers are using artificial intelligence and patient-derived heart cells to find new drugs that could treat heart damage caused by Duchenne muscular dystrophy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Greenstone Biosciences, INC. NIH-funded |
| Lab location | 1 site (Palo Alto, United States) |
| Project start | August 1, 2026 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $625,968 |
| NIH project number | 1R61HL179715-01A1 |
| Project ID | NIH-11282472 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Duchenne muscular dystrophy whose disease affects the heart and who could contribute samples or data for iPSC-derived heart cell models in future research.
✕Less likely to help
Patients without Duchenne muscular dystrophy or without heart involvement may not directly benefit from this early-stage drug discovery work.
What this research studies
This project uses patient-derived heart cell models created from stem cells to study fibrosis and dilated cardiomyopathy in Duchenne muscular dystrophy. It combines AI-driven drug screening, molecular docking, and dynamics simulations to identify promising compounds, followed by a generative AI tool that designs lead molecules. The work aims to move quickly from discovery to a screen-ready set of drug candidates that could be tested in future studies. The focus is on understanding the disease mechanisms in human-like heart tissue and pinpointing drug targets to slow or reverse heart failure in DMD patients.
Why it matters
Potential benefit: If successful, the research could yield new drug candidates to treat heart damage in people with Duchenne muscular dystrophy, potentially extending heart function and life expectancy.
How similar studies have gone: Previous studies show that iPSC-derived heart cells can model DMD-related cardiomyopathy, and AI-driven drug discovery has yielded showings in other cardiac diseases, though this exact approach is still emerging.
Where this research is happening
-
Greenstone Biosciences, INC.
Palo Alto, United States Active
Researchers
- Principal investigator: Pang, Paul — Greenstone Biosciences, INC.
- Study coordinator: Pang, Paul
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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.