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

Study Summary

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

Conditions

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

Interventions / Drugs

CTX001

View on ClinicalTrials.gov ↗

Sponsor

VRTX

Vertex Pharmaceuticals

Gene Therapy

C55/100

Study Details

Enrollment26 participants

Primary Completion—

Lead SponsorVertex Pharmaceuticals Incorporated

Data Retrieved2026-04-11 02:55:58.035170+00:00

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