Last updated on November 2019

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease


Brief description of study

This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in subjects 18 to 35 years of age 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.

Clinical Study Identifier: NCT03745287

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