Study Summary
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or 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.
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CTX001BIOLOGICAL
Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| New York Presbyterian Hospital - Morgan Stanley Children's Hospital | New York | New York | United States |
| Levine Children's Hospital - Hematology | Charlotte | North Carolina | United States |
| TriStar Medical Group Children's Specialists - Pediatric Oncology | Nashville | Tennessee | United States |
| University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology | Düsseldorf | Germany | |
| IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica | Rome | Italy | |
| King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology | Al Mathar Ash Shamali | Saudi Arabia |