Gene therapy aims to fix sickle cell disease from the inside
NCT ID NCT03282656
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tests a gene therapy approach for people with severe sickle cell disease. The treatment uses a modified virus to add a gene that boosts fetal hemoglobin, which can reduce sickling and symptoms. Ten participants will receive their own gene-corrected blood stem cells after mild chemotherapy. The goal is to see if this approach is safe and can increase fetal hemoglobin levels.
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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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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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UCLA - Mattel Children's Hospital
Los Angeles, California, 90095, United States
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Other studies related to the condition(s) this trial covers.
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