Gene therapy for Sanfilippo syndrome type B (MPS IIIB)
Official title: Development of gene therapy product for treating MPS IIIB
Funded at Neurogt, INC. · NIH-11204811 · Data updated 2026-11-12
This project aims to deliver an AAV9 gene therapy that replaces the missing NAGLU enzyme for children with MPS IIIB (Sanfilippo B).
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 | 2025 |
| Award (this year) | $1,305,826 |
| NIH project number | 4R44NS135667-02 |
| Project ID | NIH-11204811 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with a confirmed genetic diagnosis of MPS IIIB (NAGLU deficiency) who meet safety and eligibility requirements would be the primary candidates.
✕Less likely to help
People without NAGLU mutations, those with other forms of MPS, or patients with very advanced, irreversible neurological damage may not benefit from this treatment.
What this research studies
This project will develop an AAV9-based gene therapy that delivers a codon-optimized human NAGLU gene to restore the missing enzyme in people with MPS IIIB. The therapy is designed for systemic delivery that can cross the blood-brain barrier to reach both brain and body tissues and to increase enzyme production and secretion. Preclinical studies in MPS IIIB mice showed enhanced NAGLU expression and informed vector design and dosing. The overall goal is to move this product toward clinical trials and eventual availability for eligible patients.
Why it matters
Potential benefit: If successful, the therapy could restore NAGLU activity, reduce harmful glycosaminoglycan buildup, and slow or prevent neurological decline and other disease complications.
How similar studies have gone: AAV9 gene therapies have shown strong results in other neurogenetic diseases and promising results in animal models of MPS IIIB, but human clinical data specific to MPS IIIB remain limited.
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.