Developing a new platform to test treatments for Duchenne muscular dystrophy
Official title: A cross-species preclinical platform to enhance the translation of new medicines
Funded at Curi Bio INC · NIH-10983353 · Data updated 2026-11-12
This study is working on better ways to test new treatments for Duchenne muscular dystrophy (DMD) by creating models that more closely resemble how human muscles work, so we can find effective therapies for boys with this condition.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Curi Bio INC NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | December 1, 2023 |
| Project end | November 30, 2025 |
| Funding year | 2025 |
| Award (this year) | $1,149,771 |
| NIH project number | 5R44TR004557-03 |
| Project ID | NIH-10983353 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are newborn males diagnosed with Duchenne muscular dystrophy or those who are carriers of the dystrophin gene mutation.
✕Less likely to help
Patients with other forms of muscular dystrophy or unrelated genetic disorders may not benefit from this research.
What this research studies
This research focuses on creating a preclinical platform to improve the testing of new medicines for Duchenne muscular dystrophy (DMD), a severe genetic disorder affecting muscle function in newborn males. The approach involves developing advanced models that better mimic human muscle behavior, overcoming limitations of current animal models that may not accurately predict treatment outcomes. By utilizing innovative gene therapy techniques, the research aims to enhance the translation of findings from laboratory settings to real-world applications for patients.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for Duchenne muscular dystrophy, potentially improving the quality of life and longevity for affected individuals.
How similar studies have gone: Previous research has shown promise in using gene therapy approaches for DMD in animal models, but this specific preclinical platform is a novel approach.
Where this research is happening
-
Curi Bio INC
Seattle, United States Active
Researchers
- Principal investigator: Geisse, Nicholas Andrew — Curi Bio INC
- Study coordinator: Geisse, Nicholas Andrew
Related research
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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.