Gene editing frees thalassemia patients from transfusions?

NCT ID NCT06024876

First seen Feb 13, 2026 · Last updated May 14, 2026 · Updated 9 times

Summary

This early study tested a new gene therapy (CS-101) in 5 people with beta-thalassemia, a blood disorder requiring frequent transfusions. The treatment uses the patient's own stem cells, edited to produce healthy red blood cells. The main goals were safety and whether patients could stop needing transfusions for at least 6 months.

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Contacts and locations

Locations

  • The First Affiliated Hospital of Guangxi Medical University

    Nanning, China

Conditions

Explore the condition pages connected to this study.