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coagulation factor IX (Erasmus MC)
- Development stage
- Unknown
- Lead developer
- Erasmus MC
- Modality
- Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
Factor IX concentrates are a class of biologic replacement therapies used to treat and prevent bleeding episodes in patients with hemophilia B, a genetic disorder caused by a deficiency of functional coagulation factor IX. These concentrates include plasma-derived formulations (pdFIX), recombinant versions (rFIX), and extended half-life variants (EHL-FIX). By restoring levels of factor IX in the intrinsic coagulation pathway, these therapies enable the formation of thrombin and subsequent blood clots. Clinical research, such as the OPTI-CLOT TARGET study sponsored by Erasmus University Medical Center, focuses on optimizing prophylactic dosing through pharmacokinetic (PK)-guided strategies to tailor treatment to individual patient needs and activity levels.
- Other names
- factor IX concentratesFIX concentratesplasma-derived factor IXrecombinant factor IXpdFIXrFIXEHL-FIX
02
Targets
COL4 (Collagen IV)FCGRT (Neonatal crystallizable fragment receptor)F (Coagulation factors)
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