New gene therapy aims to fix sickle cell disease at its root
NCT ID NCT04819841
First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 28 times
Summary
This early-stage study tests a new gene therapy called nula-cel in 15 people with severe sickle cell disease. The treatment uses the patient's own blood stem cells to change the sickle hemoglobin into healthy adult hemoglobin. The main goals are to check safety and see if it can reduce or stop sickle cell complications.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
Contact
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Lucile Packard Children's Hospital
RECRUITINGPalo Alto, California, 94304, United States
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
Contact Email: •••••@•••••
Contact Email: •••••@•••••
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Washington University
RECRUITINGSt Louis, Missouri, 63110, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.