Muscle-targeted RNA therapeutics platform for muscular dystrophy
Official title: A Novel RNA Therapeutics Platform to Treat Facioscapulohumeral Muscular Dystrophy and other Neuromuscular Disorders
Funded at Mirecule, INC. · NIH-11393057 · Data updated 2026-11-12
A patient-friendly program aims to deliver therapeutic RNA molecules directly to muscle cells to treat inherited neuromuscular disorders like facioscapulohumeral muscular dystrophy.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mirecule, INC. NIH-funded |
| Lab location | 1 site (Gaithersburg, UNITED STATES) |
| Project start | September 20, 2021 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $608,751 |
| NIH project number | 5R44NS119147-05 |
| Project ID | NIH-11393057 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with monogenic inherited neuromuscular disorders, including facioscapulohumeral muscular dystrophy and similar conditions, who could benefit from RNA-based therapies.
✕Less likely to help
Patients with non-genetic neuromuscular conditions or those not eligible for future clinical trials may not benefit.
What this research studies
This program develops Muscle-NAV, a muscle-specific delivery system that attaches therapeutic oligonucleotides to an antibody to enter muscle cells. It focuses on diseases caused by single-gene mutations, such as certain muscular dystrophies, with the goal of correcting the faulty gene’s expression. The project uses a fast-track SBIR route to bring a promising delivery platform closer to possible therapies for patients who currently have limited treatment options. If successful, this approach could enable higher doses of RNA-based therapies to reach muscle tissue effectively.
Why it matters
Potential benefit: Could enable more effective RNA-based treatments for muscle disorders, potentially slowing disease progression and improving function.
How similar studies have gone: Other RNA delivery approaches have shown promise in preclinical models, but targeted muscle delivery remains an area of active development.
Where this research is happening
-
Mirecule, INC.
Gaithersburg, United States Active
Researchers
- Principal investigator: Saleh, Anthony D — Mirecule, INC.
- Study coordinator: Saleh, Anthony D
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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.