A new treatment approach for facioscapulohumeral muscular dystrophy and other muscle disorders
Official title: A Novel RNA Therapeutics Platform to Treat Facioscapulohumeral Muscular Dystrophy and other Neuromuscular Disorders
Funded at Mirecule, INC. · NIH-10732739 · Data updated 2026-11-12
This study is testing a new way to deliver treatments directly to muscle cells for people with inherited muscle disorders, aiming to fix the genetic problems that cause these conditions.
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 | 2024 |
| Award (this year) | $1,159,302 |
| NIH project number | 4R44NS119147-03 |
| Project ID | NIH-10732739 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with facioscapulohumeral muscular dystrophy or other inherited neuromuscular disorders caused by specific gene mutations.
✕Less likely to help
Patients with neuromuscular disorders that do not have a monogenic cause or those who do not express the targeted receptors in their muscle tissues may not benefit from this research.
What this research studies
This research focuses on developing a specialized platform called Muscle-NAVTM to deliver therapeutic oligonucleotides specifically to muscle cells affected by inherited neuromuscular disorders. By targeting the mutated genes responsible for these conditions, the therapy aims to correct the underlying genetic issues. The approach involves using antibodies that bind to muscle-specific receptors, facilitating the uptake of the therapeutic agents into the cells. Once inside, the therapy is designed to escape the endosome and reach the cytoplasm, where it can exert its effects on the mutated genes.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with facioscapulohumeral muscular dystrophy and other similar neuromuscular disorders.
How similar studies have gone: Other research has shown promise in using oligonucleotide therapeutics for genetic disorders, indicating that this approach has potential for success.
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.