Drugs / rAAV2/5-hNAGLU
rAAV2/5-hNAGLU
also known as
rAAV2/5
Gene therapy (AAV / viral vector) targets NAGLU via restoration
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT03300453 | Sep 2013 → Nov 2019 | mucopolysaccharidosis type 3B | UniQure Biopharma B.V. | Completed | No outcome recorded |
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | rAAV2/5-hNAGLU | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03300453 ↗ |
| Known as | rAAV2/5 | “with a recombinant AAV serotype 2/5 (rAAV2/5) coding human NAGLU in four children with MPS IIIB syndrome receiving immunosuppression.” PMID 34040605 ↗ May 2021 |
| Action | Restore | “To supply the missing enzyme” PMID 34040605 ↗ May 2021 |
| Modality | Gene therapy (AAV / viral vector) | “adeno-associated virus (AAV) vectors directly into the brain” PMID 34040605 ↗ May 2021 |
| Route | Other | “intracerebral administration” NCT03300453 ↗ |
| Target | NAGLU | “with a recombinant AAV serotype 2/5 (rAAV2/5) coding human NAGLU in four children with MPS IIIB syndrome receiving immunosuppression.” PMID 34040605 ↗ May 2021 |