One-Shot gene therapy aims to free patients from lifelong injections
NCT ID NCT03061201
Summary
This study tested a single-dose gene therapy called SB-525 for adults with severe hemophilia A. The goal was to see if it was safe and if it could teach the liver to produce its own missing clotting factor (Factor VIII) for years. If successful, this one-time treatment could provide lasting protection against bleeds, potentially replacing the need for lifelong, frequent intravenous treatments.
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Contacts and locations
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Locations
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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City of Hope Medical Center
Duarte, California, 91010, United States
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Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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Hemophilia Center of Western PA
Pittsburgh, Pennsylvania, 15213, United States
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Midtown Ambulatory Care Center
Sacramento, California, 95817, United States
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UC Davis Ambulatory Care Clinic
Sacramento, California, 95817, United States
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UC Davis CTSC Clinical Research Center
Sacramento, California, 95817, United States
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UC Davis Comprehensive Cancer Center
Sacramento, California, 95817, United States
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UC Davis Hemophilia Treatment Center
Sacramento, California, 95817, United States
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UC Davis Investigational Drug Services Pharmacy
Sacramento, California, 95817, United States
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UC Davis Medical Center
Sacramento, California, 95817, United States
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UPMC Montefiore Clinical and Translational Research Center
Pittsburgh, Pennsylvania, 15213, United States
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UPMC, Investigational Drug Service
Pittsburgh, Pennsylvania, 15213, United States
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USF Health Morsani Center For Advanced Healthcare
Tampa, Florida, 33612, United States
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University Of Miami Hospital and Clinics/Sylvester Comprehensive Cancer Center
Miami, Florida, 33136, United States
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University of California, San Francisco - Investigational Drug Service (IDS) Pharmacy
San Francisco, California, 94143-0622, United States
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University of California, San Francisco - Outpatient Hematology Clinic
San Francisco, California, 94143, United States
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University of California, San Francisco -Moffitt Hospital
San Francisco, California, 94143, United States
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Vanderbilt Hemostasis Treatment Clinic
Nashville, Tennessee, 37232, United States
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Vanderbilt Hemostasis-Thrombosis Clinic
Nashville, Tennessee, 37232, United States
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Vanderbilt University Medical Center Clinical Research Center
Nashville, Tennessee, 37212-8646, United States
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Washington Institute for Coagulation
Seattle, Washington, 98101, United States
Conditions
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