Gene-edited stem cells: a step toward transfusion-free life for β-thalassemia?

NCT ID NCT06479616

Recruiting now Disease control Sponsor: Children's Hospital of Fudan University Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study follows 5 people with β-thalassemia who received CS-101, a gene-edited stem cell therapy, in an earlier trial. Researchers will monitor them for years to see if the treatment remains safe and allows them to stay free from blood transfusions. The goal is to understand whether this one-time therapy can provide lasting control of the disease.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: CS-101 (a gene-edited stem cell therapy)
What this could lead to: If successful, this could show that a single treatment with CS-101 allows people with β-thalassemia to live without needing regular blood transfusions.
What could go wrong: This is a very small follow-up study (5 people) with no control group, so results may not apply to everyone. Long-term risks like cancer or blood disorders are still being checked.

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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.

BETA-THALASSEMIA

Contacts and locations

Locations

Children's Hospital of Fudan University
RECRUITING

Shanghai, Shanghai Municipality, 201102, China

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