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Gene-Editing breakthrough offers hope for blood disorder patients

NCT ID NCT06024876

First seen Feb 13, 2026 · Last updated May 23, 2026 · Updated 11 times

Summary

This early-phase study tested a new gene-edited stem cell treatment (CS-101) in 5 people with beta-thalassemia, a severe blood disorder requiring regular blood transfusions. The goal was to see if the treatment is safe and can help patients produce healthy blood cells, potentially reducing or eliminating the need for transfusions. Participants received their own genetically modified stem cells after a conditioning regimen.

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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.