The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).
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Key Inclusion Criteria: * Participants with documented βS/βC (HbSC) genotype * Participants must be eligible for autologous stem cell transplant as per investigator's judgment Key Exclusion Criteria: * A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement * Participants with prior hematopoietic stem cell transplant (HSCT) * Treatment with regular RBC transfusions that, in the opinion of the investigator, cannot be interrupted after engraftment. Other protocol defined Inclusion/Exclusion criteria may apply.