Table 1.

Gene therapy and gene editing clinical trials for β-thalassemia

Trial identifier	Phase/status	Enrolled patients	Myeloablative regimen	Mobilization protocol	Drug product	Sponsor/center
NCT02453477 [25]	Phase 1–2 Active, not recruiting	TDT 9 (3 adults, 6 children)	Treosulfan and Thiotepa	G-CSF and Plerixafor	Autologous HSCs genetically modified with GLOBE lentiviral vector encoding for the human β-globin gene	IRCCS San Raffaele Fondazione Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
NCT01745120 (HGB-204) [24]	Phase 1–2 Completed	TM 18 (between 12 and 35 years of age)	Busulfan	G-CSF and Plerixafor	Autologous HSCs transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene	Bluebird bio 6 international sites
NCT02151526 (HGB-205) [24]	Phase 1–2 Completed	TDT 4 (between 5 and 35 years of age)	Busulfan (adjusted based on daily PK monitoring)	G-CSF and Plerixafor (after 3 months of enhanced transfusion)	Autologous HSCs transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene	Bluebird bio Necker Children’s Hospital, Paris, France
NCT02906202 (HGB 207)	Phase 3 Active, not recruiting	TDT non β0/β0,  ≤ 50 years of age Estimated enrollment: 23 pts	Busulfan	G-CSF and Plerixafor	Autologous HSCs transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene	Bluebird bio 8 international sites
NCT03207009 (HGB 212)	Phase 3 Recruiting	TDT β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 ≤ 50 years of age Estimated enrollment: 18 pts	Busulfan	G-CSF and Plerixafor	Utologous HSCs transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene	Bluebird bio 9 international sites
NCT03745287	Phase 1–2 Recruiting	Estimated enrollment: 45 participants including SCD and other hematological disorders	Busulfan	NA	CTX001: autologous CD34 + HSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene	Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics 12 international sites
NCT03432364	Phase 1–2 Recruiting	TDT  ≥ 18 and ≤ 40 years of age Estimated enrollment: 45 participants	Busulfan	NA	ST-400: autologous CD34 + HSPCs genetically modified with ZFN technology at the erythroid-specific enhancer of the BCL11A gene	Sangamo Therapeutics 6 sites in the United States

TDT transfusion dependent thalassemia, TM thalassemia major, G-CSF granulocyte-colony stimulating factor, PK pharmacokinetics, SCD sickle cell disease, NA not available, hHSPCs Human Hematopoietic Stem and Progenitor Cells, ZFN zinc finger nuclease