One-Time gene therapy aims to free patients from lifelong injections
NCT ID NCT06611436
Summary
This early-stage study is testing a new, one-time gene therapy called BE-101 for adults with moderately severe or severe hemophilia B. The goal is to see if a single infusion of a patient's own genetically edited cells can safely help the body produce its own missing clotting factor (Factor IX), potentially reducing or eliminating the need for regular replacement injections. The study will first test increasing doses in small groups to find a safe and effective level.
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Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
Locations
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University of California, Davis
RECRUITINGDavis, California, 95616, United States
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University of Michigan
RECRUITINGAnn Arbor, Michigan, 48109, United States
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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55455, United States
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Washington Center for Bleeding Disorders
RECRUITINGSeattle, Washington, 98101, United States
Conditions
Explore the condition pages connected to this study.