Drugs / VOLRUBIGENE RALAPARVOVEC
VOLRUBIGENE RALAPARVOVEC
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT03223194 | Sep 2017 → Feb 2021 | Crigler-Najjar syndrome | Audentes Therapeutics | Terminated | No outcome recorded Stop: Business |
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | VOLRUBIGENE RALAPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298182 ↗ |
| Known as | AT-003 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298182 ↗ |
| Known as | AT342 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03223194 ↗ |
| Known as | Volrubigen ralaparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298182 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “AT342 is an AAV8 vector containing a functional copy of the UGT1A1 gene.” NCT03223194 ↗ |
| Route | Intravenous | “Subjects will receive a single dose of AT342 delivered intravenously.” NCT03223194 ↗ |
| Target | UGT1A1 | “AT342 is an AAV8 vector containing a functional copy of the UGT1A1 gene.” NCT03223194 ↗ |