A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
Latest Information Update: 07 Sep 2025
At a glance
- Drugs Exagamglogene autotemcel (Primary) ; Busulfan; Plerixafor
- Indications Sickle cell anaemia
- Focus Adverse reactions; Proof of concept; Registrational; Therapeutic Use
- Acronyms CLIMB-121
- Sponsors Vertex Pharmaceuticals
Most Recent Events
- 11 Aug 2025 Status changed from active, no longer recruiting to completed.
- 31 Oct 2024 Planned End Date changed from 1 Oct 2024 to 1 Jul 2025.
- 31 Oct 2024 Planned primary completion date changed from 1 Oct 2024 to 1 Jul 2025.