Split-Intein Gene Delivery to Treat Duchenne Muscular Dystrophy
Official title: Split-Intein Protein Ligation: Enabling AAV Delivery of Large Genes for Clinical Translation in Genetic Cardiac Diseases
Funded at Kinea Bio, INC. · NIH-11410482 · Data updated 2026-11-12
A new gene therapy approach aims to deliver a larger, more functional dystrophin gene to heart and muscle cells in people with Duchenne muscular dystrophy.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Kinea Bio, INC. NIH-funded |
| Lab location | 1 site (Mukilteo, UNITED STATES) |
| Project start | September 1, 2026 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $599,669 |
| NIH project number | 1R44HL182563-01A1 |
| Project ID | NIH-11410482 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Duchenne muscular dystrophy who have cardiomyopathy or are at risk for cardiac involvement and could participate in preclinical testing that informs future human trials.
✕Less likely to help
Patients without DMD or without cardiac involvement may not benefit from this specific therapy.
What this research studies
This project tests a novel gene therapy technique that uses dual AAV vectors to deliver a bigger dystrophin gene and restore heart and skeletal muscle function in Duchenne patients. Researchers will assess where the therapy goes in the heart, how much dystrophin is produced, and whether it improves heart performance in dystrophin-deficient rats. They will also generate cardiomyocytes from human iPSCs to see if the treatment corrects disease features in human cells, and build a functional potency assay using 3D engineered heart tissue from patients. The goal is to create a viable, more effective treatment pathway that could translate to patients in the future.
Why it matters
Potential benefit: If successful, this therapy could provide stronger, more lasting restoration of heart and muscle function for people with DMD.
How similar studies have gone: Prior gene therapies using smaller dystrophin constructs have shown partial benefits; this approach seeks to overcome size limits with a novel dual-vector strategy.
Where this research is happening
-
Kinea Bio, INC.
Mukilteo, United States Active
Researchers
- Principal investigator: Childers, Martin K — Kinea Bio, INC.
- Study coordinator: Childers, Martin K
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.