Drugs / VOXERALGAGENE AUTOTEMCEL
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT03454893 | Feb 2018 → Mar 2022 | Fabry disease | AVROBIO | Terminated | No outcome recorded |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | VOXERALGAGENE AUTOTEMCEL | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095471 ↗ |
| Known as | AVR-RD-01 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03454893 ↗ |
| Action | Restore | “results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.” NCT03454893 ↗ |
| Route | Intravenous | “Single IV infusion” NCT03454893 ↗ |