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