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
Latest Information Update: 08 Apr 2025
At a glance
- Drugs Exagamglogene autotemcel (Primary)
- Indications Beta-thalassaemia; Sickle cell anaemia
- Focus Registrational; Therapeutic Use
- Sponsors Vertex Pharmaceuticals
Most Recent Events
- 03 Apr 2025 Planned End Date changed from 1 Feb 2025 to 9 Jun 2027.
- 03 Apr 2025 Planned primary completion date changed from 1 Feb 2025 to 9 Jun 2027.
- 11 Feb 2025 According to Vertex Pharmaceuticals media release,the company expects to complete dosing of children 5 to 11 years of age group in 2025.