Gene editing offers hope for blood disorder patients

NCT ID NCT06328764

First seen Feb 10, 2026 · Last updated May 14, 2026 · Updated 15 times

Summary

This early-stage study tests a new gene-edited stem cell treatment (CS-101) for people with beta-thalassemia, a severe blood disorder requiring frequent transfusions. Ten participants aged 6 to 35 will receive their own genetically modified stem cells to see if it can safely reduce or eliminate the need for blood transfusions. The main goals are checking safety and whether patients can go without transfusions for at least six months.

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Contacts and locations

Locations

  • The First Affiliated Hospital of Guangxi Medical University

    Nanning, Guangxi, China

Conditions

Explore the condition pages connected to this study.