Gene therapy aims to stop sickle cell pain crises
NCT ID NCT07432867
First seen Feb 28, 2026 · Last updated Jun 20, 2026 · Updated 13 times
Summary
This trial tests a new gene therapy called DREAM01 for people with severe sickle cell disease who cannot find a matching bone marrow donor. The therapy uses the patient's own blood stem cells, modified to produce healthy hemoglobin and block the sickle cell gene. Up to 15 participants aged 12-35 will receive a single infusion, with the goal of reducing painful vaso-occlusive events and long-term organ damage.
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the original study
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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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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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Department of Biotherapy, Necker-Enfants Malades Hospital
RECRUITINGParis, Île-de-France Region, 75015, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
DREAM01 gene therapy (modified stem cells)
What this could lead to
If successful, this could offer a one-time treatment that reduces or eliminates painful crises and organ damage for people with severe sickle cell disease who lack a matched donor.
What could go wrong
This is an early phase 1/2 trial with only 15 participants, so results may not apply to everyone. Gene therapy carries risks like failed engraftment or side effects from the conditioning regimen.
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.