Ongoing gene-addition trials
| Clinical trial . | Clinicaltrials.gov registry no. . | Phase . | Mechanism . | Vector . | Cell source . | Conditioning regimen . | Status . |
|---|---|---|---|---|---|---|---|
| HGB-206 | NCT02140554 | 1/2 | βA-T87Q addition | Lentiviral | Bone marrow; plerixafor mobilized | Busulfan | Active, not recruiting |
| HGB-210 | NCT04293185 | 3 | βA-T87Q addition | Lentiviral | Bone marrow; plerixafor mobilized | Busulfan | Recruiting |
| ARU-1801 | NCT02186418 | 1/2 | γ Addition | Lentiviral | Bone marrow; plerixafor mobilized | Melphalan | Recruiting |
| Gene transfer for sickle cell disease | NCT03282656 | 1 | γ Induction via addition of shRNA silencing BCL11A | Lentiviral | Plerixafor mobilized | Busulfan | Recruiting |
| DREPAGLOBE | NCT03964792 | 1/2 | βAS3 Addition | Lentiviral | Bone marrow; plerixafor mobilized | — | Not yet recruiting |
| Stem cell gene therapy for sickle cell disease | NCT02247843 | 1/2 | βAS3 Addition | Lentiviral | Plerixafor mobilized | Busulfan | Recruiting |
| CSL200 gene therapy in adults with severe sickle cell disease | NCT04091737 | 1 | γG16D Addition | Lentiviral | Plerixafor mobilized | Melphalan | Active, not recruiting |
| Clinical trial . | Clinicaltrials.gov registry no. . | Phase . | Mechanism . | Vector . | Cell source . | Conditioning regimen . | Status . |
|---|---|---|---|---|---|---|---|
| HGB-206 | NCT02140554 | 1/2 | βA-T87Q addition | Lentiviral | Bone marrow; plerixafor mobilized | Busulfan | Active, not recruiting |
| HGB-210 | NCT04293185 | 3 | βA-T87Q addition | Lentiviral | Bone marrow; plerixafor mobilized | Busulfan | Recruiting |
| ARU-1801 | NCT02186418 | 1/2 | γ Addition | Lentiviral | Bone marrow; plerixafor mobilized | Melphalan | Recruiting |
| Gene transfer for sickle cell disease | NCT03282656 | 1 | γ Induction via addition of shRNA silencing BCL11A | Lentiviral | Plerixafor mobilized | Busulfan | Recruiting |
| DREPAGLOBE | NCT03964792 | 1/2 | βAS3 Addition | Lentiviral | Bone marrow; plerixafor mobilized | — | Not yet recruiting |
| Stem cell gene therapy for sickle cell disease | NCT02247843 | 1/2 | βAS3 Addition | Lentiviral | Plerixafor mobilized | Busulfan | Recruiting |
| CSL200 gene therapy in adults with severe sickle cell disease | NCT04091737 | 1 | γG16D Addition | Lentiviral | Plerixafor mobilized | Melphalan | Active, not recruiting |