Membrane-repair protein treatment for Becker muscular dystrophy
Official title: Membrane repair as a therapeutic intervention for treating Becker Muscular Dystrophy
Funded at Myofinity Biosciences INC · NIH-11196200 · Data updated 2026-11-12
This project is developing a modified protein medicine to help muscle cells patch their damaged outer membrane in people with Becker muscular dystrophy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Myofinity Biosciences INC NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 23, 2023 |
| Project end | August 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $587,105 |
| NIH project number | 5R42AR083329-03 |
| Project ID | NIH-11196200 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with a confirmed diagnosis of Becker muscular dystrophy and ongoing muscle weakness would be the likely candidates for future trials of this therapy.
✕Less likely to help
People without Becker muscular dystrophy or those whose muscle problems are caused by unrelated conditions are unlikely to benefit from this treatment.
What this research studies
The team is engineering a version of the MG53 protein that helps muscle cell membranes heal after injury and changing its sequence to reduce potential toxicity. They will make the protein under controlled manufacturing methods and run the studies needed to apply to the FDA for first-in-human testing. The work builds on animal data showing MG53 can improve muscle pathology in dystrophin-deficient models but focuses on a safer, optimized therapeutic for people with Becker muscular dystrophy. The project is led by a biotech company in Boston and is part of a combined Phase I/II SBIR effort to move the product toward clinical trials.
Why it matters
Potential benefit: If successful, the treatment could lower muscle damage and improve strength or slow decline in people with Becker muscular dystrophy.
How similar studies have gone: Related recombinant MG53 protein improved muscle pathology in dystrophin-deficient mouse models, but this engineered version is new and still needs safety and human testing.
Where this research is happening
-
Myofinity Biosciences INC
Boston, United States Active
Researchers
- Principal investigator: Weisleder, Noah — Myofinity Biosciences INC
- Study coordinator: Weisleder, Noah
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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.