Drugs / AAV2/8-HLP-FVIII-V3
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT03328130 | Nov 2017 → Jun 2025 | retinitis pigmentosa | eyeDNA Therapeutics | Terminated | No outcome recorded Stop: Business |
| Phase 1/2 | NCT03173521 | Jul 2017 → Jul 2021 | mucopolysaccharidosis type 6 | Fondazione Telethon | Completed | Met primary |
| Phase 1/2 | NCT03001830 | Jun 2017 → Jan 2029 expected | hemophilia A | University College, London | Active not recruiting | No outcome recorded |
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AAV2/8-HLP-FVIII-V3 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03001830 ↗ |
| Known as | AAV2/5-hPDE6B | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03328130 ↗ |
| Known as | AAV2/8.TBG.hARSB | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03173521 ↗ |
| Action | Restore | “AAV2/8-HLP-FVIII-V3 leads to long-term, endogenous expression of FVIII” NCT03001830 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “adeno-associated viral vector” NCT03001830 ↗ |
| Route | Other | “single systemic administration of AAV2/8-HLP-FVIII-V3” NCT03001830 ↗ |
| Target | PDE6B | “supply to the target cells the PDE6B therapeutic gene” NCT03328130 ↗ |