Drug Information

Drug General Information
Drug ID
D00TXL
Former ID
DIB001147
Drug Name
AAV2-hFIX16
Synonyms
AAV-F.IX, University of Pennsylvania; AAV-factor IX gene therapy (hemophilia B), University of Pennsylvania
Indication Factor IX deficiency [ICD10:D67] Phase 1 [522086]
Company
University of Pennsylvania
Target and Pathway
Target(s) Coagulation factor IX Target Info Modulator [544166]
KEGG Pathway Complement and coagulation cascades
PANTHER Pathway Blood coagulation
PathWhiz Pathway Coagulation
Reactome Extrinsic Pathway of Fibrin Clot Formation
Intrinsic Pathway of Fibrin Clot Formation
Gamma-carboxylation of protein precursors
Transport of gamma-carboxylated protein precursors from the endoplasmic reticulum to the Golgi apparatus
Removal of aminoterminal propeptides from gamma-carboxylated proteins
WikiPathways Complement and Coagulation Cascades
PTM: gamma carboxylation, hypusine formation and arylsulfatase activation
Blood Clotting Cascade
Formation of Fibrin Clot (Clotting Cascade)
References
Ref 522086ClinicalTrials.gov (NCT00515710) LTFU for Gene Transfer Subjects With Hemophilia B. U.S. National Institutes of Health.
Ref 544166Assessing the potential for AAV vector genotoxicity in a murine model. Correction in: Blood. 2011 June 16; 117(24): 6739.

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