Gene therapy hope for kids with blood disorders
NCT ID NCT07190001
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 24 times
Summary
This early-stage study tests a single dose of a gene therapy called YOLT-204 in 18 children (ages 3-17) with severe beta-thalassemia or sickle cell disease. The goal is to see if it is safe and can raise fetal hemoglobin levels enough to reduce or stop the need for regular blood transfusions. Participants will be followed for up to 15 years to monitor long-term effects.
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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.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Guangzhou women and children's medical center
Guangzhou, Guangdong, 510405, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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