New gene therapy could stop sickle cell pain crises

NCT ID NCT07432867

First seen Feb 28, 2026 · Last updated Apr 28, 2026 · Updated 7 times

Summary

This study tests a new gene therapy for people with severe sickle cell disease who do not have a matching bone marrow donor. The treatment uses the patient's own blood stem cells, which are modified in a lab to produce healthy red blood cells and reduce sickling. The goal is to decrease painful blockages in blood vessels and prevent long-term organ damage. The study involves 15 participants aged 12 to 35 and will monitor safety and effectiveness over time.

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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.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Department of Biotherapy, Necker-Enfants Malades Hospital

    RECRUITING

    Paris, Île-de-France Region, 75015, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.