Table 1 Open sickle cell gene therapy... | American Society of Hematology

Table 1

Open sickle cell gene therapy trials

Group	Vector*		Conditioning	Enrollment	ClinicalTrials.gov identifier	Status
Group	Gene cassette	Insulator	Conditioning	Enrollment	ClinicalTrials.gov identifier	Status
Bluebird Bio	β^A-T87Q-globin	None	Busulfan: 12.8 mg/kg IV, pK adjusted	Ages 5-37 y, up to 7 subjects; adult, up to 8 subjects	NCT02140554; NCT02151526	Ongoing: at least 1 patient treated⁶⁷
University of California, Los Angeles	βAS3-globin	FB†	Busulfan: 12.8 mg/kg IV, pK adjusted	Adult, up to 6 subjects	NCT02247843	Open
Cincinnati Children’s Hospital Medical Center	γ-globin	None	Melphalan: 140 mg/m²	Adult to age 35 y, up to 10 subjects	NCT02186418	Open

Group	Vector*		Conditioning	Enrollment	ClinicalTrials.gov identifier	Status
Group	Gene cassette	Insulator	Conditioning	Enrollment	ClinicalTrials.gov identifier	Status
Bluebird Bio	β^A-T87Q-globin	None	Busulfan: 12.8 mg/kg IV, pK adjusted	Ages 5-37 y, up to 7 subjects; adult, up to 8 subjects	NCT02140554; NCT02151526	Ongoing: at least 1 patient treated⁶⁷
University of California, Los Angeles	βAS3-globin	FB†	Busulfan: 12.8 mg/kg IV, pK adjusted	Adult, up to 6 subjects	NCT02247843	Open
Cincinnati Children’s Hospital Medical Center	γ-globin	None	Melphalan: 140 mg/m²	Adult to age 35 y, up to 10 subjects	NCT02186418	Open

All vectors described are SIN lentiviruses.

Enhancer blocker only.