Table 7.

Quantification/surrogates of engrafted corrected HSCs proposed for use in measuring cure in SCD

End pointMeasuresGrade of Cure
GT/E VCN or edited cells and alleles in infusion product and 0.5, 1, 2, 5, 10, and 15 y after genetic modification (%); genetically modified and allele modified CFUc (%) VCN in infused product and up to 6 mo after GT/E can be higher; stable VCN (at year 1 onward) that results in: 
1: 100% genetically corrected RBCs 
2: 75%-100% corrected RBCs 
3: 50%-75% corrected RBCs 
4: 25%-50% corrected RBCs 
5: 0%-25% corrected RBCs. 
Corrected RBCs may be defined as antisickling Hb containing RBCs and/or RBCs that resist sickling (%) 
Engraftment Normal or genetically modified HSC chimerism (%) 1: >50% 
2: >20% 
End pointMeasuresGrade of Cure
GT/E VCN or edited cells and alleles in infusion product and 0.5, 1, 2, 5, 10, and 15 y after genetic modification (%); genetically modified and allele modified CFUc (%) VCN in infused product and up to 6 mo after GT/E can be higher; stable VCN (at year 1 onward) that results in: 
1: 100% genetically corrected RBCs 
2: 75%-100% corrected RBCs 
3: 50%-75% corrected RBCs 
4: 25%-50% corrected RBCs 
5: 0%-25% corrected RBCs. 
Corrected RBCs may be defined as antisickling Hb containing RBCs and/or RBCs that resist sickling (%) 
Engraftment Normal or genetically modified HSC chimerism (%) 1: >50% 
2: >20% 

CFUc, colony-forming units in culture.

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