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.
Study on Clazakizumab for Heart Disease in Adults with End-Stage Kidney Disease on Dialysis
AustriaEfficacy and safety of human alpha1‑proteinase inhibitor weekly infusions in adults with alpha1 antitrypsin deficiency–related emphysema
Denmark
BelgiumStudy of Etranacogene Dezaparvovec (CSL222) in Adolescent Males with Severe or Moderately Severe Hemophilia B
AustriaStudy on the Safety and Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Adults with Severe or Moderately Severe Hemophilia B and AAV5 Antibodies
BulgariaStudy of Human Normal Immunoglobulin in Adults with Secondary Immune Deficiency after CAR T‑cell or T‑cell Redirecting Therapy for Hematologic Malignancies
BelgiumA study to evaluate the effectiveness and safety of vamifeport in adults with hereditary hemochromatosis
AustriaStudy of Nebulized Human Normal Immunoglobulin (CSL787) for Adults with Non-cystic Fibrosis Bronchiectasis to Find Best Dose and Test Safety
BelgiumStudy on the Effects of Human Normal Immunoglobulin in Adults with Dermatomyositis
BelgiumSafety Study of Switching from Current Treatment to Garadacimab in Patients Age 12 and Older with Hereditary Angioedema
GermanyStudy on the Effectiveness of Human Normal Immunoglobulin for Adults with Post-COVID-19 Postural Orthostatic Tachycardia Syndrome (POTS)
GermanyStudy on the Safety and Effectiveness of CSL889 for Adults and Adolescents with Sickle Cell Disease During Painful Crises
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