Gene-edited stem cells: a step toward transfusion-free life for β-thalassemia?
NCT ID NCT06479616
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.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for BETA-THALASSEMIA are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Children's Hospital of Fudan University
RECRUITINGShanghai, Shanghai Municipality, 201102, China
Contact Phone: •••-•••-•••• Email: •••••@•••••