Developing a gene therapy for Sanfilippo Syndrome type B using a modified AAV8 vector
Official title: Procession to IND of a capsid mutated AAV8 codon optimized NAGLU vector for treatment of Sanfilippo Syndrome type B
Funded at University of Florida · NIH-10902765 · Data updated 2026-11-12
This study is testing a new gene therapy designed to help people with Sanfilippo Syndrome type B by delivering a missing enzyme directly into the brain, and it's aimed at making sure the treatment is safe and works well before it can be used in humans.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | July 9, 2024 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $583,430 |
| NIH project number | 1UG3NS137533-01 |
| Project ID | NIH-10902765 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Sanfilippo Syndrome type B, particularly children who are in the early stages of the disease.
✕Less likely to help
Patients with other types of Sanfilippo Syndrome or those who are not genetically predisposed to the NAGLU deficiency may not benefit from this research.
What this research studies
This research focuses on creating a gene therapy using a modified AAV8 vector to treat Sanfilippo Syndrome type B, a genetic disorder that affects the brain and leads to severe neurological issues. The approach involves injecting the therapy into the brain or cerebrospinal fluid to deliver the NAGLU enzyme, which is deficient in patients with this condition. The study will assess the effectiveness and safety of the therapy through various animal models, including mice and non-human primates, to ensure it can correct lysosomal storage issues and improve overall health outcomes. Additionally, the research will involve optimizing the production process to ensure the therapy is safe and effective for potential human use.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with Sanfilippo Syndrome type B, potentially improving their quality of life and lifespan.
How similar studies have gone: Previous research has shown promise in using gene therapy approaches for similar lysosomal storage disorders, indicating potential for success in this novel application.
Where this research is happening
-
University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Heldermon, Coy D — University of Florida
- Study coordinator: Heldermon, Coy D
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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.