7 United States sites, 1 Belgium site, 1 Canada site, 1 Germany site, 1 Italy site
18 to 35 Years
βS/βS
Phase 1/Phase 2
This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in up to 12
subjects 18 to 35 years of age 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.
– Subjects ≥ 18 and ≤ 35 years of age
– Diagnosis of severe sickle cell disease as defined by:
– Documented βS/βS genotype
– History of at least two severe vaso-occlusive crisis events per year for the previous
two years prior to enrollment
– Eligible for autologous stem cell transplant as per investigators judgment
Key
– An available 10/10 human leukocyte antigen (HLA)-matched related donor
– Prior hematopoietic stem cell transplant (HSCT)
– Clinically significant and active bacterial, viral, fungal, or parasitic infection
Other protocol defined inclusion/exclusion criteria may apply