Gene-Editing breakthrough? early trial tackles sickle cell at its root
NCT ID NCT03653247
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 22 times
Summary
This early-stage study tested a new gene-edited stem cell treatment (BIVV003) in 7 adults with severe sickle cell disease. The goal was to see if the treatment is safe and can help the body make healthy red blood cells. Participants received their own genetically modified stem cells after a conditioning regimen, and researchers tracked survival, engraftment, and side effects.
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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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Locations
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Investigational Site Number 101
Bethesda, Maryland, 20892, United States
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Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
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UCSF Benioff Children's Hospital
Oakland, California, 94609, United States
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University of California Davis Comprehensive Cancer Center
Sacramento, California, 95817, United States
Conditions
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