Drugs / RAG1 LV CD34+ cells
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04797260 | Jul 2021 → Dec 2031 | recombinase activating gene 1 deficiency | Videja B.V. | Suspended | No outcome recorded Stop: Enrollment |
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 | RAG1 LV CD34+ cells | “Patients will be infused with autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (RAG1 LV CD34+ cells).” NCT04797260 ↗ |
| Action | Restore | “gene therapy using lentiviral (LV) self-inactivating (SIN) vectors expressing codon-optimized human RAG1 in a mouse model for RAG1-deficient SCID effectively restores T and B...” NCT04797260 ↗ |
| Modality | Cell therapy | “autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector” NCT04797260 ↗ |
| Target | RAG1 | “lentiviral vectors with different internal promoters driving codon-optimized RAG1” PMID 32322605 ↗ Mar 2020 |