Drugs / AVR-RD-02
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT04145037 | May 2019 → Aug 2023 | Gaucher disease type I | AVROBIO | Terminated | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT05815004 | Oct 2023 → Dec 2027 | Gaucher disease type III | AVROBIO | Withdrawn | No outcome recorded |
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AVR-RD-02 | “AVR-RD-02 Drug product: active substance is autologous CD34+ enriched hematopoietic stem cells (HSCs) that have been genetically modified ex vivo with a lentiviral vector (LV)...” NCT05815004 ↗ |
| Action | Restore | “genetically modified ex vivo with a lentiviral vector (LV) to contain a ribonucleic acid (RNA) transcript that, after reverse transcription, results in codon-optimized,...” NCT04145037 ↗ |