Drugs / rAAV9-DES-hGAA
Trials 1 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT02240407 | Oct 2017 → Aug 2021 | glycogen storage disease II | University of Florida | Completed | No outcome recorded |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | rAAV9-DES-hGAA | “rAAV9-DES-hGAA injected intramuscularly into the TA” NCT02240407 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “A recombinant AAV vector has been generated” NCT02240407 ↗ |
| Route | Intramuscular | “rAAV9-DES-hGAA injected intramuscularly into the TA” NCT02240407 ↗ |
| Target | GAA | “carry the codon-optimized acid alpha-glucosidase (coGAA) gene” NCT02240407 ↗ |