. Author manuscript; available in PMC: 2021 Jul 21.

Published in final edited form as: N Engl J Med. 2020 Dec 5;384(3):205–215. doi: 10.1056/NEJMoa2029392

Table 1.

Baseline Characteristics of the Treated Patients and the Drug Product.^*

Patient Number	Age in Yr, Sex	Genotype	Severe Symptoms	Receiving Transfusion, Hydroxyurea	CD34+ Cell Dose	VCN of Product	CD34+ Cells Transduced	Follow-up
					cells per kg	copies per diploid genome	%	mo
2	20, M	β^S/β^S	Previous stroke	Yes, No	5.1	3.3	95.8	29
3	25, M	β^S/β^S	Previous stroke	Yes, No	6.7	5.0	98.6	19
4	24, M	β^S/β^S	Priapism	No, Yes	5.2	3.3	95.5	20
6	16, F	β^S/β⁰	Vaso-occlusive event, ACS	No, Yes	8.3	6.9	100.0	16
7	12, F	β^S/β^S	Previous stroke	Yes, No	6.1	2.8	93.1	12
8	7, M	β^S/β^S	Vaso-occlusive event	No, Yes	4.9	1.8	62.0	7

Baseline laboratory values represent the most recent samples collected before cell infusion for which all relevant data are available. ACS denotes acute chest syndrome, and VCN vector copy number.