Developing a gene therapy to treat MPS IIIB in children
Official title: Development of gene therapy product for treating MPS IIIB
Funded at Neurogt, INC. · NIH-10921942 · Data updated 2026-11-12
This study is working on a new gene therapy to help children with Mucopolysaccharidosis IIIB (MPS IIIB) by using a special virus to deliver a corrected gene that can help produce an important enzyme they are missing, with the hope of making them feel better and reducing the effects of the disease.
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, 2025 |
| Funding year | 2024 |
| Award (this year) | $456,414 |
| NIH project number | 1R44NS135667-01A1 |
| Project ID | NIH-10921942 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children aged 0-11 years diagnosed with MPS IIIB.
✕Less likely to help
Patients with other types of mucopolysaccharidosis or those outside the specified age range may not benefit from this research.
What this research studies
This research focuses on creating a gene therapy product aimed at treating Mucopolysaccharidosis IIIB (MPS IIIB), a rare genetic disorder affecting children. The approach involves using an adeno-associated virus (AAV) vector to deliver a corrected version of the gene responsible for producing an essential enzyme that is deficient in patients with MPS IIIB. By targeting the underlying genetic defect, the therapy aims to improve enzyme levels and reduce the harmful effects of the disease. The research includes preclinical testing in animal models to assess the safety and effectiveness of the therapy before moving towards clinical application.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for children suffering from MPS IIIB, potentially improving their quality of life and extending their lifespan.
How similar studies have gone: Previous research has shown success with AAV gene therapy in treating other lysosomal storage diseases, indicating a promising approach for MPS IIIB.
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.