Casgevy 4 - 13 x 10^6 cells/mL dispersion for infusion
Sponsors
Vertex Pharmaceuticals Inc.
Conditions
Severe sickle cell disease (SCD)Sickle Cell DiseaseTransfusion dependent thalassemiaTransfusion-Dependent-ThalassemiaTransfusion-dependent β-thalassemia (TDT)
Phase 1
Phase 3
A Long-term Follow-up Study of Subjects with β-Thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)
RecruitingCTIS2024-512654-19-00
Start: 2020-03-06Target: 37Updated: 2025-11-17
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
RecruitingCTIS2024-514641-12-00
Start: 2022-10-26Target: 6Updated: 2025-08-08
A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in subjects with Transfusion-Dependent β-Thalassemia
CompletedCTIS2024-516894-57-00
Start: 2018-09-07End: 2025-10-10Target: 5Updated: 2025-08-08
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease
RecruitingCTIS2024-513978-22-00
Start: 2022-07-20Target: 4Updated: 2025-12-23
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia
RecruitingCTIS2024-513349-35-00
Start: 2022-05-16Target: 4Updated: 2026-01-06