Single gene shot could tame hemophilia b bleeding
NCT ID NCT01687608
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a single intravenous dose of a gene therapy called AskBio009 in up to 30 adults with severe hemophilia B. The therapy delivers a working copy of the factor IX gene to help the body produce its own clotting factor. The main goal is to check safety, but researchers will also measure whether it raises factor IX levels and reduces bleeding episodes.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AskBio009 (BAX 335) gene therapy
What this could lead to
If successful, this could provide long-term control of hemophilia B with a single injection, reducing or eliminating the need for regular factor IX infusions.
What could go wrong
This is an early phase 1/2 trial with only 30 participants, so results may not apply to everyone. Gene therapy can cause immune reactions or may not raise factor IX levels enough to stop bleeding.
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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BloodCenter of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Bloodworks Northwest
Seattle, Washington, 98104, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Boston
Boston, Massachusetts, 022105, United States
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Emory University
Atlanta, Georgia, 30322, United States
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Gulf States Hemophilia and Thrombosis Center
Houston, Texas, 77030, United States
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Mount Sinai Medical Center
New York, New York, 10029, United States
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Orthopaedic Hemophilia Treatment Center
Los Angeles, California, 90007, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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The Hemophilia Center, Oregon Health and Science University
Portland, Oregon, 97239, United States
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U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center
Aurora, Colorado, 80045, United States
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University of California Davis Medical Center
Sacramento, California, 95817, United States
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University of California at San Diego Medical Center
San Diego, California, 92103-8651, United States
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University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute
Minneapolis, Minnesota, 55455, United States