Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05477563 · clinicaltrials.gov ↗

PHASE3

Phase

RECRUITING

Status

26

Enrollment

INDUSTRY

Sponsor class

Conditions

Beta-Thalassemia
Thalassemia
Hematologic Diseases
Genetic Diseases, Inborn
Hemoglobinopathies
Sickle Cell Disease
Sickle Cell Anemia

Interventions

BIOLOGICAL: CTX001

Sponsor

Vertex Pharmaceuticals Incorporated