Age

12–50

Sex

ALL

Healthy volunteers

Not accepted

≥12 to ≤ 40 years
Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures:
recurrent severe VOC (≥ 4 episodes in the preceding 2 years)
ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year)
Lansky/Karnofsky performance status of ≥ 80

Available 10/10 HLA-matched sibling donor
Prior HSCT or gene therapy
Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder
Clinically significant and active bacterial, viral, fungal or parasitic infection
Pregnancy or breastfeeding in a postpartum female
Presence of a chromosomal abnormality/mutation that may put the participant at an increased risk for MDS or AML per investigator's judgment

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease