Study Summary
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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Exa-celBIOLOGICAL
Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan.
Study Locations
No locations listed.