AAV gene therapy for treating MPS IIIB in children
Official title: Development of gene therapy product for treating MPS IIIB
Funded at Neurogt, INC. · NIH-11406160 · Data updated 2026-11-12
A small, fast-track NIH grant is funding a pediatric gene therapy approach to treat MPS IIIB using an AAV9 vector to deliver the NAGLU enzyme.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Neurogt, INC. NIH-funded |
| Lab location | 1 site (Chapel Hill, UNITED STATES) |
| Project start | September 1, 2024 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $1,341,983 |
| NIH project number | 5R44NS135667-03 |
| Project ID | NIH-11406160 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children aged 0-11 with MPS IIIB or related lysosomal storage disease presenting suitable for AAV9-based gene therapy approaches.
✕Less likely to help
Patients with advanced disease where delivery to target tissues is unlikely to yield clinical benefit may not experience meaningful improvement.
What this research studies
This project aims to translate a gene therapy using AAV9 to deliver a codon-optimized NAGLU gene for MPS IIIB from the lab toward clinical use. The plan includes preclinical work and steps toward human trials to help children who have this rare lysosomal storage disease. Researchers are focusing on overcoming delivery barriers and ensuring safety while aiming for meaningful reduction of disease-causing storage in affected organs. If successful, the therapy could address the root cause of the disorder and potentially slow or halt disease progression. The SBIR format emphasizes rapid development toward commercialization.
Why it matters
Potential benefit: Could provide a one-time or limited-treatment option that addresses the underlying cause of MPS IIIB and reduces organ storage of harmful substances.
How similar studies have gone: Prior work with systemic AAV9 delivery for neurogenetic diseases has shown reproducible safety and efficacy signals in animal models, supporting further development.
Where this research is happening
-
Neurogt, INC.
Chapel Hill, United States Active
Researchers
- Principal investigator: Mccarty, Douglas M — Neurogt, INC.
- Study coordinator: Mccarty, Douglas M
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.