Developing a Gene Therapy for Sanfilippo Syndrome Type B
Procession to IND of a capsid mutated AAV8 codon optimized NAGLU vector for treatment of Sanfilippo Syndrome type B
This project is working to prepare a new gene therapy to treat Sanfilippo Syndrome Type B, aiming to correct the underlying genetic problem.
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 ID | NIH-11135541 on NIH RePORTER |
What this research studies
Our goal is to advance a special gene therapy, called AAVtcm8-coNAGLU, closer to being tested in people with Sanfilippo Syndrome Type B. This therapy uses a modified virus to deliver a working gene that can help the body produce a missing enzyme. We are performing detailed tests in animal models, including mice, dogs, and non-human primates, to ensure the therapy is safe and effective. These steps are crucial for getting approval from the FDA to begin clinical trials in patients.
Who could benefit from this research
Good fit: This research is focused on developing a treatment for individuals diagnosed with Sanfilippo Syndrome Type B.
Not a fit: Patients with other genetic conditions or those not diagnosed with Sanfilippo Syndrome Type B would not directly benefit from this specific therapy.
Why it matters
Potential benefit: If successful, this gene therapy could offer a way to correct the genetic defect in Sanfilippo Syndrome Type B, potentially improving symptoms and quality of life for patients.
How similar studies have performed: Gene therapies for other genetic disorders have shown promise, and this approach builds on previous successful preclinical work in animal models for Sanfilippo Syndrome Type B.
Where this research is happening
Gainesville, United States
- University of Florida — Gainesville, United States (Active)
Researchers
- Principal investigator: Heldermon, Coy D — University of Florida
- Study coordinator: Heldermon, Coy D
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 below.