Gene-Editing hope: could BIVV003 free sickle cell patients from pain crises?
NCT ID NCT03653247
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This early-phase trial tested a new gene therapy called BIVV003 in 7 adults with severe sickle cell disease. The treatment uses the patient's own stem cells, which are gene-edited in a lab and then infused back after chemotherapy. The main goals were to check safety and see if the cells engraft successfully. Because it's a small, early study, results are preliminary and more research is needed.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
BIVV003 (gene-edited stem cells)
What this could lead to
If successful, this could point toward a one-time treatment that reduces or eliminates sickle cell crises without needing a donor.
What could go wrong
This is a very early, small study (only 7 people) focused on safety. The gene-editing approach is new, and long-term effects or failure to control the disease are possible.
Disclaimer
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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.
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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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
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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