Genome editing and gene therapy clinical trials in SCD as of March 2021
| Goal . | Nuclease/target . | Sponsor, collaborator . | Clinical trial ID . | Estimated participants . |
|---|---|---|---|---|
| Elevate HbF | CTX001/BCL11A | Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics | NCT03745287 | 45 |
| Elevate HbF | Plerixafor/BCL11A | Bioverativ, a Sanofi company | NCT03653247 | 8 |
| Elevate HbF | OTQ923 or HIX763/BCL11A | Novartis Pharmaceuticals | NCT04443907 | 30 |
| Goal . | Nuclease/target . | Sponsor, collaborator . | Clinical trial ID . | Estimated participants . |
|---|---|---|---|---|
| Elevate HbF | CTX001/BCL11A | Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics | NCT03745287 | 45 |
| Elevate HbF | Plerixafor/BCL11A | Bioverativ, a Sanofi company | NCT03653247 | 8 |
| Elevate HbF | OTQ923 or HIX763/BCL11A | Novartis Pharmaceuticals | NCT04443907 | 30 |
| Goal . | Viral vector . | Sponsor, collaborator . | Clinical trial ID . | Estimated participants . |
|---|---|---|---|---|
| Repair HbS mutation | Lenti/G-βAS3-FB lentiviral Vector | California Institute for Regenerative Medicine | NCT02247843 | 6 |
| Elevate HbF | ARU-1801 | Aruvant Sciences GmbH | NCT02186418 | 10 |
| Repair HbS mutation | GLOBE1 lentiviral vector expressing the βAS3 globin gene | Assistance Publique–Hôpitaux de Paris | NCT03964792 | 10 |
| Repair HbS mutation | LentiGlobin BB305 lentiviral vector | bluebird bio | NCT04293185 | 35 |
| Repair HbS mutation | Lentiviral vector encoding the normal β-globin gene | Memorial Sloan Kettering Cancer Center, Sanofi | NCT02193191 | 39 |
| Repair HbS mutation | LentiGlobin BB305 lentiviral vector | bluebird bio | NCT02140554 | 50 |
| Elevate HbF | Lentiviral vector containing a short hairpin RNA targeting BCL11A | Boston Children’s Hospital | NCT03282656 | 15 |
| Goal . | Viral vector . | Sponsor, collaborator . | Clinical trial ID . | Estimated participants . |
|---|---|---|---|---|
| Repair HbS mutation | Lenti/G-βAS3-FB lentiviral Vector | California Institute for Regenerative Medicine | NCT02247843 | 6 |
| Elevate HbF | ARU-1801 | Aruvant Sciences GmbH | NCT02186418 | 10 |
| Repair HbS mutation | GLOBE1 lentiviral vector expressing the βAS3 globin gene | Assistance Publique–Hôpitaux de Paris | NCT03964792 | 10 |
| Repair HbS mutation | LentiGlobin BB305 lentiviral vector | bluebird bio | NCT04293185 | 35 |
| Repair HbS mutation | Lentiviral vector encoding the normal β-globin gene | Memorial Sloan Kettering Cancer Center, Sanofi | NCT02193191 | 39 |
| Repair HbS mutation | LentiGlobin BB305 lentiviral vector | bluebird bio | NCT02140554 | 50 |
| Elevate HbF | Lentiviral vector containing a short hairpin RNA targeting BCL11A | Boston Children’s Hospital | NCT03282656 | 15 |