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.

NCT: NCT03745287

Locations: University of Illinois at Chicago Hospitals and Health Systems

Link: https://clinicaltrials.gov/ct2/show/NCT03745287?recrs=ad&cond=Sickle+Cell+Anemia&locn=University+of+Chicago&cntry=US&age=0&draw=2&rank=8