Abstract
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with
severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of
autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs)
using CTX001.
