Can gene therapy free sickle cell patients from painful crises?
NCT ID NCT07752043
First seen Aug 07, 2026 · Last updated Aug 07, 2026
Summary
This trial compares a new gene therapy to standard care in people aged 12 to 35 with severe sickle cell disease. The gene therapy uses a patient's own blood stem cells, modified to produce a therapeutic form of hemoglobin and reduce the sickling hemoglobin. The study measures how well each approach prevents vaso-occlusive crises, reduces mortality, and improves overall health and quality of life.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Gene-modified autologous CD34+ cells transduced with a lentiviral vector expressing therapeutic beta-globin and an anti-HbS miRNA, compared with standard care (hydroxyurea, transfusions, supportive care).
- What this could lead to
- If successful, this gene therapy could reduce or eliminate painful vaso-occlusive crises and improve quality of life for people with severe sickle cell disease, potentially offering a one-time treatment alternative to lifelong standard care.
- What could go wrong
- This is an early-phase trial with a small number of participants, so results may not be conclusive. Gene therapy carries risks such as immune reactions, failure of engraftment, or long-term side effects that are not yet fully known.
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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
Locations
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Necker-Enfants Malades Hospital, Apheresis Unit
Paris, Île-de-France Region, 75015, France
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