Advancing manufacturing and release testing for a Duchenne gene editing therapy
Official title: Advancing manufacturing and release testing of a gene editing therapy for Duchenne muscular dystrophy
Funded at Myogene Bio, LLC · NIH-11526941 · Data updated 2026-11-12
A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Myogene Bio, LLC NIH-funded |
| Lab location | 1 site (San Diego, UNITED STATES) |
| Project start | September 1, 2025 |
| Project end | July 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $498,365 |
| NIH project number | 5SB1AR084953-02 |
| Project ID | NIH-11526941 on NIH RePORTER |
Who could benefit from this research
✓Could help
Not applicable to this grant as described; this is a manufacturing/CMC development effort.
✕Less likely to help
This project does not involve patient participation or direct patient care.
What this research studies
This grant focuses on improving the manufacturing process and analytical tests for a two-vector AAV gene editing therapy aimed at Duchenne muscular dystrophy. researchers will run a Design of Experiments to boost yield and quality, and will develop product-specific analytics and standard non-compendial assays for both vectors. The work includes collaboration with a contract development and manufacturing organization and regulatory consultants to ensure compliance with regulatory standards. The goal is to finalize standard operating procedures for manufacturing and release testing to support progression toward clinical material and IND filing.
Why it matters
Potential benefit: If successful, the project could streamline production and quality testing for a Duchenne gene editing therapy, helping accelerate clinical development and potential patient access.
How similar studies have gone: Previous DoE-driven process optimizations have improved consistency in biologics manufacturing, but this exact dual-vector AAV gene editing approach is in early stages.
Where this research is happening
-
Myogene Bio, LLC
San Diego, United States Active
Researchers
- Principal investigator: Young, Courtney S — Myogene Bio, LLC
- Study coordinator: Young, Courtney S
Related research
Other NIH-funded projects on the same topics.
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Understanding why Duchenne Muscular Dystrophy worsens and how to stop it
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-
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This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.
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A wearable-sensor project aims to quietly track and quantify how motor skills develop in babies and young boys with Duchenne Muscular Dystrophy.
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Genetic factors that influence Duchenne Muscular Dystrophy progression
A study to find genes that change how severe Duchenne muscular dystrophy is in boys and young men, using patient data and DNA samples.
-
Optimizing gene therapy to help breathing in Duchenne muscular dystrophy
A Duke University team is testing a gene therapy approach to improve breathing in boys with Duchenne muscular dystrophy.
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.