Ongoing gene-editing trials
| Clinical trial . | Clinicaltrials.gov registry no. . | Phase . | Mechanism . | Gene editor . | Cell source . | Conditioning regimen . | Status . |
|---|---|---|---|---|---|---|---|
| CLIMB-SCD-121 | NCT03745287 | 1/2 | Erythroid lineage-specific enhancer of the BCL11A disruption | CRISPR-Cas9 | Plerixafor mobilized | Busulfan | Recruiting |
| PRECIZN-1 | NCT03653247 | 1/2 | BCL11A locus targeting | Zinc finger nuclease | Plerixafor mobilized | Busulfan | Recruiting |
| Clinical trial . | Clinicaltrials.gov registry no. . | Phase . | Mechanism . | Gene editor . | Cell source . | Conditioning regimen . | Status . |
|---|---|---|---|---|---|---|---|
| CLIMB-SCD-121 | NCT03745287 | 1/2 | Erythroid lineage-specific enhancer of the BCL11A disruption | CRISPR-Cas9 | Plerixafor mobilized | Busulfan | Recruiting |
| PRECIZN-1 | NCT03653247 | 1/2 | BCL11A locus targeting | Zinc finger nuclease | Plerixafor mobilized | Busulfan | Recruiting |