TRANSFUSION-DEPENDENT THALASSEMIA

Clinical trials for TRANSFUSION-DEPENDENT THALASSEMIA explained in plain language.

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Trials to join now! 1 Not yet recruiting 2 Not yet finished but already full! 3 Completed 3

CRISPR stem cell therapy could end transfusions for sickle cell and thalassemia patients

Disease control Recruiting now

This study tests a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or s…

Matched conditions: TRANSFUSION-DEPENDENT THALASSEMIA

Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control

Last updated Jun 27, 2026 12:00 UTC

TRANSFUSION-DEPENDENT THALASSEMIA

Get alerted when new TRANSFUSION-DEPENDENT THALASSEMIA trials appear

CRISPR stem cell therapy could end transfusions for sickle cell and thalassemia patients