Creating a new treatment for a genetic neurological disorder using specialized vectors.
Official title: MANUFACTURING OF PLASMID AND VECTOR AND GLP DOSE-ESCALATION TOXICOLOGY STUDIES IN NORMAL RATS AND NORMAL NHPS
Funded at Battelle Centers/pub Hlth Res & Evaluatn · NIH-10948246 · Data updated 2026-11-12
This study is working on a new treatment for Aspartylglucosaminuria (AGU) by creating a special delivery system for a gene that could help, and they’re testing it in animals first to make sure it’s safe and effective before trying it in people.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Battelle Centers/pub Hlth Res & Evaluatn NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project start | September 30, 2023 |
| Project end | September 29, 2026 |
| Funding year | 2023 |
| Award (this year) | $2,086,305 |
| NIH project number | 75N95022D00021-0-759502300003-1 |
| Project ID | NIH-10948246 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Aspartylglucosaminuria or those at risk of developing this genetic disorder.
✕Less likely to help
Patients with other neurological disorders or those not affected by Aspartylglucosaminuria may not benefit from this research.
What this research studies
This research focuses on developing a new therapy for Aspartylglucosaminuria (AGU), a severe genetic neurological disorder. The team will manufacture a specific vector and plasmids that carry the AGA gene, which is crucial for potential treatment. They will conduct dose-escalation toxicology studies in normal rats and non-human primates to ensure the safety and effectiveness of the therapy before it can be tested in humans. The research aims to establish a reliable manufacturing process and conduct thorough testing to support future clinical trials.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment for patients suffering from Aspartylglucosaminuria.
How similar studies have gone: Similar approaches in gene therapy have shown promise in treating genetic disorders, indicating potential success for this novel treatment.
Where this research is happening
-
Battelle Centers/pub Hlth Res & Evaluatn
Columbus, United States Active
Researchers
- Principal investigator: Burnaugh, Amanda — Battelle Centers/pub Hlth Res & Evaluatn
- Study coordinator: Burnaugh, Amanda
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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.