Trans-BBB AAV9 gene therapy for CNS symptoms in kids with MPS I
Official title: Trans-BBB AAV9 gene therapy targeting CNS neuropathy of MPS I
Funded at Neurogt, INC. · NIH-11417197 · Data updated 2026-11-12
A gene therapy designed to get an IDUA enzyme into the brain to help children with MPS I who have brain-related problems.
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 16, 2025 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $1,484,525 |
| NIH project number | 5R44NS143501-02 |
| Project ID | NIH-11417197 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with MPS I, particularly those with neurological involvement, may be candidates for future trials of this gene therapy.
✕Less likely to help
Patients without CNS involvement or those not fitting the trial’s specific delivery and safety criteria may not benefit.
What this research studies
This project aims to advance a gene replacement therapy that can cross the blood-brain barrier and deliver the missing enzyme to the CNS in children with MPS I. The team uses a self-complementary AAV9 vector to target neurological symptoms and aims to move toward clinical use and commercialization. The work involves preclinical development focused on safety, biodistribution, and efficacy in a young patient population. If successful, it could address neurological and cardio-respiratory complications that current treatments do not fully treat.
Why it matters
Potential benefit: If successful, the therapy could reduce brain and nerve-related damage in children with MPS I and improve neurodevelopmental outcomes.
How similar studies have gone: Previous studies suggest rAAV9-based approaches can cross the BBB and address neurological aspects in lysosomal storage diseases, but this exact therapy is still in 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
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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.