Drugs / autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein
autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein
Cell therapy targets BCL11A
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT07708350 | Aug 2026 → Dec 2028 expected | sickle cell disease | Daniel Bauer | Not yet recruiting | No outcome recorded |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07708350 ↗ |
| Modality | Cell therapy | “autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein” NCT07708350 ↗ |
| Route | Intravenous | “The edited cells will be infused intravenously.” NCT07708350 ↗ |
| Target | BCL11A | “BCL11A enhancer targeting Cas9 ribonucleoprotein” NCT07708350 ↗ |