Table 2.

A summary of HSC gene-editing therapy trials in SCD and TDT.

Trial	Phase	Year	Study Drug	Target Gene	HSC Source	Conditioning	Sponsor	Location	Reference
Sickle cell disease
NCT04774536	1/2	2021	CRISPR/Cas9: CRISPR_SCD001	β-globin	BM	Myeloablative Busulfan	University of California, San Francisco	USA	N/A
NCT04853576	1/2	2021	CRISPR/Cas12: EDIT-301	BCL11A ESE	Plerixafor mobilization	Myeloablative busulfan	Editas Medicine	USA	N/A
NCT04819841	1/2	2021	CRISPR/Cas9: GPH101	β-globin	BM	Myeloablative busulfan	Graphite Bio	USA	N/A
NCT05145062	N/A	2021	ZFN: BIVV003	BCL11A ESE	Plerixafor mobilization	Myeloablative busulfan	Bioverativ	USA	[142]
NCT04443907	1/2	2020	CRISPR/Cas9: OTQ923 / HIX763	BCL11A ESE	N/A	N/A	Novartis Pharmaceuticals	USA	N/A
NCT03653247	1/2	2019	ZFN: BIVV003	BCL11A ESE	Plerixafor mobilization	Myeloablative busulfan	Bioverativ	USA	[142]
NCT04208529	N/A	2019	CRISPR/Cas9: CTX001	BCL11A ESE	BM	Myeloablative busulfan	Vertex Pharmaceuticals	USA	[144]
NCT03745287	2/3	2018	CRISPR/Cas9: CTX001	BCL11A ESE	BM	Myeloablative busulfan	Vertex Pharmaceuticals	USA	[144]
β-Thalassemia
NCT04205435	1/2	2021	CRISPR/Cas9	β-globin	N/A	N/A	Biorary Laboratories	China	N/A
NCT04208529	N/A	2021	CRISPR/Cas9: CTX001	BCL11A ESE	BM	Myeloablative busulfan	Vertex Pharmaceuticals	USA	[144]
NCT04211480	1/2	2020	CRISPR/Cas9	BCL11A ESE	N/A	N/A	Biorary Laboratories	China	N/A
NCT03655678	2/3	2018	CRISPR/Cas9: CTX001	BCL11A ESE	BM	Myeloablative busulfan	Vertex Pharmaceuticals	USA, Canada, Germany, Italy, UK	[144]
NCT03432364	1/2	2018	ZFN: ST-400	BCL11A ESE	G-CSF & plerixafor mobilization	Myeloablative busulfan	Sangamo Therapeutics	USA	[141]

ESE: erythroid-specific enhancer, BM: bone marrow, RIC: reduced-intensity conditioning, N/A: not applicable.