Sickle cell gene therapy trial pulled before it even started
NCT ID NCT05951205
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study was designed to test a single dose of exa-cel, a gene-edited cell therapy, in people with severe sickle cell disease who have the HbSC genotype. Participants would have received their own modified stem cells after chemotherapy. However, the trial was withdrawn before any patients were enrolled, so no data on safety or effectiveness were collected.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Exa-cel (also called Exagamglogene autotemcel or CTX001), a gene-edited cell therapy made from the patient's own blood stem cells
What this could lead to
If it worked, this could provide a one-time treatment to reduce or eliminate sickle cell crises for people with the HbSC genotype.
What could go wrong
The trial was withdrawn before enrolling anyone, so no results exist. Gene-editing therapies can have serious side effects, including infertility and blood cancers, and require intensive chemotherapy beforehand.
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.