A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease

drug protocol number

VX21-CTX001-161

drug sponsor

Vertex Pharmaceuticals

drug study

Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001)

drug trial site

King Faisal Specialist Hospital and Research Center (Riyadh)

drug status

Ongoing

drug phase