Gene therapy aims to fix hemophilia b at the source
NCT ID NCT03961243
First seen Apr 24, 2026 · Last updated Jun 19, 2026 · Updated 8 times
Summary
This early-stage trial tests a gene therapy for hemophilia B, a bleeding disorder. Ten patients will receive their own stem cells modified with a working gene for clotting factor IX. The goal is to see if it's safe and can help the body produce its own clotting factor, potentially reducing bleeds.
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This is a summary of
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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Lentiviral factor IX gene modified stem cells
What this could lead to
If successful, this could reduce or eliminate the need for regular factor IX infusions in hemophilia B patients.
What could go wrong
This is a very early Phase I trial with only 10 participants. It may not work, and there are risks like immune reactions or the gene not lasting.
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.