Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

Protocol: 
AAAR9004
Phase: 
NA

Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

Evaluate the safety and efficacy of a single dose of autologous CRISPR-Cas9
modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs)
(CTX001) in subjects with severe sickle cell disease (SCD)

Are you Eligible? (Inclusion Criteria)

1. Are you 18 to 35 years old?
2. Does you have severe sickle cell disease?
3. Have you been hospitalized or went to the emergency room for your pain?
4. Have you been told you were eligible for a stem cell transplant?

Trial Location

Herbert Irving Comprehensive Cancer Center
161 Fort Washington Ave.
Herbert Irving Pavilion
New York, NY 10032
United States