Rare diseases
Investigational molecules
Locations
1020 1st Avenue, 19406-1310, United States
Research activity includes haemophilia B and Hemophilia B, with a strong emphasis on AAV-based gene therapy, factor IX expression, and long-term control of bleeding risk in adolescent and adult populations.
Clinical interest also extends to neutralizing antibodies and sustained treatment outcomes after gene-based intervention.
CSL Behring LLC runs clinical trials in conditions such as Factor IX deficiency, Hereditary angioedema, Arteriosclerosis, Alpha-1 antitrypsin deficiency, among others.
Clinical trials run by this sponsor – those enrolling participants first.
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