Gene-Editing hope for sickle cell patients, but trial halted early
NCT ID NCT04443907
First seen Jan 11, 2026 · Last updated May 14, 2026 · Updated 19 times
Summary
This early-stage study tested a gene-edited stem cell treatment (OTQ923) in 4 people with severe sickle cell disease. The goal was to boost fetal hemoglobin to reduce painful crises and other complications. The trial was terminated early, so full results are limited, but it aimed to see if the treatment was safe and could help control the disease long-term.
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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.
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Locations
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Memorial Sloan Kettering Cancer Ctr
New York, New York, 10065, United States
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St Jude Children's Research Hospital
Memphis, Tennessee, 38105-3678, United States
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University of Chicago
Chicago, Illinois, 60637, United States
Conditions
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