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

Who May Qualify: - ≥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 Who Should NOT Join This Trial: - Available 10/10 HLA-matched sibling donor - Prior HSCT or gene therapy - Prior or current malignancy or myeloproliferative or a significant coagulation or weakened immune system 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 Always talk to your doctor about whether this trial is right for you.