Muscle-targeted RNA therapy for FSHD and other inherited muscle disorders
Official title: A Novel RNA Therapeutics Platform to Treat Facioscapulohumeral Muscular Dystrophy and other Neuromuscular Disorders
Funded at Mirecule, INC. · NIH-11194285 · Data updated 2026-11-12
This project is creating a way to deliver RNA medicines directly into muscle for people with facioscapulohumeral muscular dystrophy (FSHD) and other inherited muscle diseases.
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 | 2025 |
| Award (this year) | $1,225,173 |
| NIH project number | 5R44NS119147-04 |
| Project ID | NIH-11194285 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with a confirmed diagnosis of FSHD or other single-gene inherited muscle disorders who are potential candidates for antisense or RNA-based therapies would be most relevant.
✕Less likely to help
People with non-genetic or immune-mediated muscle conditions, those without a targetable gene change, or individuals with very advanced muscle loss may not benefit from this specific approach.
What this research studies
The team is building a muscle-specific delivery platform called Muscle-NAV that links an antibody to an antisense RNA drug so the medicine binds a muscle receptor and is taken into muscle cells. The antibody guides uptake and the company’s conjugation chemistry aims to help the RNA escape intracellular compartments to reach the cell’s cytoplasm. Researchers will test the approach in laboratory models and animals to see if it reduces toxic gene activity such as DUX4 in FSHD and protects muscle cells. If the platform works, the same delivery system could be adapted to many other single-gene muscle disorders.
Why it matters
Potential benefit: If successful, this could reduce toxic DUX4 levels in FSHD and enable effective RNA-based treatments that preserve muscle function across multiple inherited muscle diseases.
How similar studies have gone: Antisense RNA drugs have helped some neuromuscular diseases (for example nusinersen in SMA), but effective, targeted delivery to skeletal muscle remains largely unproven, so this platform is promising but experimental.
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.