Gene editing offers hope for blood disorder patients

NCT ID NCT06024876

First seen Jun 27, 2026 ยท Last updated Jun 27, 2026

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 red 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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Conditions

The condition(s) this trial relates to.

beta thalassemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • The First Affiliated Hospital of Guangxi Medical University

    Nanning, China