Table 3.
Gene therapy trials for β-thalassemia
| Therapy | Gene modification technique | Genetic mechanism | Phase of development | Also in development for sickle cell disease? | Comments |
|---|---|---|---|---|---|
| Betibeglogene autotemcel (beti-cel; formerly BB305)8-11,17,18 | Lentiviral vector | Insertion of modified β-globin | Phase 3 trials | Yes | Reopened after temporary halt due to AML cases in sickle cell patients |
| GLOBE12 | Lentiviral vector | Insertion of human β-globin | Phase 1/2 trial completed | No | Intrabone administration |
| CTX00114,15 | CRISPR-Cas9 | Targets BCL11A enhancer → HbF induction | Phase 1/2 trial | Yes | |
| ST-40016 | ZFN | Targets BCL11A enhancer → HbF induction | Phase 1/2 trial | No |