Study Summary
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).
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CRISPR_SCD001DRUG
CRISPR\_SCD001 is administered by IV infusion following myeloablative conditioning with busulfan.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| University of California, Los Angeles | Los Angeles | California | United States |
| UCSF Benioff Children's Hospital | Oakland | California | United States |