MyoPAXon cell transplant to restore muscle in Duchenne muscular dystrophy
Systemic Transplantation of MyoPAXon: IND Enabling Studies for the Treatment of DMD
This project plans to develop a cell therapy called MyoPAXon to replace damaged muscles in people with Duchenne muscular dystrophy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Myogenica INC. NIH-funded |
| Lab location | 1 site (Saint Paul, United States) |
| Project ID | NIH-11001285 on NIH RePORTER |
What this research studies
This project is developing MyoPAXon, an off-the-shelf muscle-forming cell product made from induced pluripotent stem cells, to replace damaged skeletal muscle in Duchenne muscular dystrophy. Researchers are performing IND-enabling preclinical work including safety, biodistribution, and efficacy testing in animal models to prepare for human trials. The team intends to deliver the cells systemically so they can reach many muscles and create new muscle fibers and muscle stem cells. Currently the work is preclinical and not open for patient enrollment, but it is designed to lead to future clinical trials.
Who could benefit from this research
Good fit: Ideal candidates would be people with Duchenne muscular dystrophy caused by mutations in the dystrophin gene, particularly those with progressive muscle weakness who may qualify for future cell therapy trials.
Not a fit: People without Duchenne muscular dystrophy, those with end-stage heart or lung failure, or anyone not eligible for cell therapy would likely not benefit from this program.
Why it matters
Potential benefit: If successful, MyoPAXon could rebuild weakened muscles, improve strength and mobility, and slow disease progression in people with DMD.
How similar studies have performed: Related gene and cell therapy approaches have shown promise in animal models and some early human trials, but systemic iPSC-derived muscle cell transplants remain largely untested in people.
Where this research is happening
Saint Paul, United States
- Myogenica INC. — Saint Paul, United States (Active)
Researchers
- Principal investigator: Kyba, Michael — Myogenica INC.
- Study coordinator: Kyba, Michael
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.