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Target intelligence / Profile preview
Coagulation Factor IX Padua Variant (R338L) (FIX Padua)
- Target
- FIX Padua
- Molecular classification
- Serine protease, Coagulation factor
01
Overview
The F9 Padua variant (R338L) is a gain-of-function mutant of coagulation factor IX (FIX), a serine protease essential for blood clotting. The mutation enhances FIX activity, making it a highly effective therapeutic transgene for hemophilia B gene therapy. It allows for lower vector doses and potentially reduces immunogenicity. Gene therapies based on this variant aim to restore endogenous FIX production, improving hemostatic control.
- Other names
- Factor IX PaduaF9 PaduaR338L FIXR338L Factor IX
02
Mechanism of action
Recombinant hyperactive coagulation factor IX increases the rate of the intrinsic pathway of the coagulation cascade, promoting thrombin formation and improving hemostasis.
03
Biological functions
Blood coagulationHemostasis
04
Disease associations
Hemophilia BThrombophilia (rare, in native form)
05
Safety considerations
Thrombosis (potential risk, carefully managed via dosing)Immunogenicity (potential for anti-FIX antibodies)
06
Biomarkers
Factor IX activity levelsPresence of anti-FIX antibodies
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