Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D00TXL
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| Former ID |
DIB001147
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| Drug Name |
AAV2-hFIX16
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| Synonyms |
AAV-F.IX, University of Pennsylvania; AAV-factor IX gene therapy (hemophilia B), University of Pennsylvania
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| Indication | Factor IX deficiency [ICD-11: 3B11; ICD-10: D67] | Phase 1 | [1] | |
| Company |
University of Pennsylvania
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Coagulation factor IX (F9) | Target Info | Modulator | [2] |
| 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 | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT00515710) LTFU for Gene Transfer Subjects With Hemophilia B. U.S. National Institutes of Health. | |||
| REF 2 | Assessing the potential for AAV vector genotoxicity in a murine model. Correction in: Blood. 2011 June 16; 117(24): 6739. | |||
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