Sickle cell gene therapy trial halted after just 4 patients
NCT ID NCT04443907
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial tested a gene-edited stem cell product called OTQ923 in 4 people with severe sickle cell disease. The goal was to boost fetal hemoglobin to reduce painful crises and other complications. The study was terminated early, so we have limited data on safety and effectiveness.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
OTQ923 (genome-edited stem cells)
What this could lead to
If successful, this approach could point toward a one-time treatment that reduces painful crises and other severe complications of sickle cell disease.
What could go wrong
This was a very early, small trial (only 4 participants) that was terminated, so results are limited. Gene-editing therapies also carry risks like failed engraftment or unintended genetic changes.
Disclaimer
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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.
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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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
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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