Gene therapy breakthrough? new treatment aims to fix sickle cell at the source
NCT ID NCT04819841
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial is testing a new gene therapy called nula-cel for people with severe sickle cell disease. The therapy modifies a patient's own blood stem cells to produce normal hemoglobin instead of sickle hemoglobin. About 15 participants aged 12 to 40 will receive the treatment after a conditioning regimen. The main goals are to check safety and see if the therapy can reduce or eliminate sickle cell complications.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
nula-cel (a gene therapy that modifies a patient's own blood stem cells to produce normal hemoglobin)
What this could lead to
If it works, this could provide a one-time treatment that controls severe sickle cell disease by enabling the body to make healthy hemoglobin instead of sickle hemoglobin.
What could go wrong
This is a very early, first-in-human trial with only 15 participants. The conditioning regimen has serious risks, and the gene therapy may not work as expected or could cause side effects. Long-term safety and effectiveness are unknown.
Disclaimer
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the original study
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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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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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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
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Lucile Packard Children's Hospital
RECRUITINGPalo Alto, California, 94304, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Email: •••••@•••••
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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: •••••@•••••