A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2 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.
Key Inclusion Criteria:

 -  Diagnosis of severe sickle cell disease as defined by:

 -  Documented severe sickle cell disease 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 Exclusion Criteria:

 -  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