Gene-Editing hope: could a One-Time transplant fix sickle cell?
NCT ID NCT03653247
First seen Nov 01, 2025 · Last updated May 11, 2026 · Updated 21 times
Summary
This early-stage study tested a new treatment called BIVV003 for adults with severe sickle cell disease. The approach uses a person's own blood stem cells, which are gene-edited in a lab and then given back after chemotherapy. The main goals were to see if the treatment is safe and if the new cells start making healthy blood. Only 7 people took part, so results are very preliminary.
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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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