Creating a new treatment for a genetic neurological disorder using AAV vectors
Official title: MANUFACTURING OF PLASMID AND VECTOR AND GLP DOSE-ESCALATION TOXICOLOGY STUDIES
Funded at Battelle Centers/pub Hlth Res & Evaluatn · NIH-11172202 · Data updated 2026-11-12
This study is working on a new treatment for Aspartylglucosaminuria, a serious genetic brain disorder, using a special delivery system to help make sure it’s safe and effective for patients who currently have limited options.
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 | 2024 |
| Award (this year) | $131,706 |
| NIH project number | 75N95022D00021-P00001-759502300003-1 |
| Project ID | NIH-11172202 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Aspartylglucosaminuria or related genetic neurological disorders.
✕Less likely to help
Patients with other unrelated neurological disorders or those not diagnosed with Aspartylglucosaminuria may not benefit from this research.
What this research studies
This research focuses on developing an AAV9 vector and plasmids as a potential therapy for Aspartylglucosaminuria, a severe genetic neurological disorder. The project involves manufacturing the vector and conducting Good Laboratory Practice (GLP) dose-escalation toxicology studies to ensure safety and efficacy. Patients may benefit from this innovative approach as it aims to provide a new treatment option for a currently underserved condition. The research will include rigorous testing to confirm the quality and effectiveness of the manufactured products.
Why it matters
Potential benefit: If successful, this research could lead to a new therapeutic option for patients suffering from Aspartylglucosaminuria.
How similar studies have gone: Similar approaches using AAV vectors have shown promise in other genetic therapies, indicating potential for success in this novel application.
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
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.