AAV9 gene therapy for Aspartylglucosaminuria (AGU)
IND-enabling studies for Aspartylglucosaminuria (AGU) to support the initiation of an AAV9/AGA gene transfer clinical trial
This project is preparing an AAV9-based gene therapy to deliver a working AGA gene for children with Aspartylglucosaminuria (AGU).
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ut Southwestern Medical Center NIH-funded |
| Lab location | 1 site (Dallas, United States) |
| Project ID | NIH-11169696 on NIH RePORTER |
What this research studies
If I or my child had AGU, this project would be doing the lab and animal safety tests needed to move a gene therapy into humans. Researchers will produce an AAV9 viral vector carrying a healthy AGA gene, run assays to measure toxic substrate levels, and perform preclinical safety and dosing studies. The plan includes intrathecal delivery (injection into spinal fluid) to reach the brain and builds on prior FDA discussions to prepare an IND application. If those steps are completed, the team aims to open a Phase I/II clinical trial where eligible patients could receive the therapy and be followed for safety and benefit.
Who could benefit from this research
Good fit: Ideal candidates would be people with genetically confirmed AGU, especially children with early-stage disease who meet safety criteria and can travel to the trial site.
Not a fit: People with very advanced, late-stage disease, significant medical contraindications, or who cannot undergo intrathecal delivery may not receive benefit from this approach.
Why it matters
Potential benefit: If successful, the therapy could lower the toxic buildup that drives brain damage in AGU and slow or prevent worsening of developmental and neurological problems.
How similar studies have performed: AAV9 delivered intrathecally has been used in other rare pediatric neurodegenerative disorders and progressed to early human trials, providing precedent though AGU-specific results are still experimental.
Where this research is happening
Dallas, United States
- Ut Southwestern Medical Center — Dallas, United States (Active)
Researchers
- Principal investigator: Gray, Steven J — Ut Southwestern Medical Center
- Study coordinator: Gray, Steven J
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.