Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DX0NW9
|
|||
| Drug Name |
TAK-754
|
|||
| Synonyms |
BAX 888
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|
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| Drug Type |
Gene therapy
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| Indication | Haemophilia A [ICD-11: 3B10.0; ICD-9: 286] | Phase 1/2 | [1] | |
| Company |
Takeda
|
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Coagulation factor VIII (F8) | Target Info | . | [2] |
| KEGG Pathway | Complement and coagulation cascades | |||
| Panther Pathway | Blood coagulation | |||
| Pathwhiz Pathway | Coagulation | |||
| Reactome | Platelet degranulation | |||
| Intrinsic Pathway of Fibrin Clot Formation | ||||
| Common Pathway of Fibrin Clot Formation | ||||
| COPII (Coat Protein 2) Mediated Vesicle Transport | ||||
| Cargo concentration in the ER | ||||
| WikiPathways | Complement and Coagulation Cascades | |||
| Blood Clotting Cascade | ||||
| Formation of Fibrin Clot (Clotting Cascade) | ||||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03370172) A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion. U.S.National Institutes of Health. | |||
| REF 2 | A reprieve from hemophilia A, but for how long? Nat Biotechnol. 2020 Oct;38(10):1107-1109. | |||
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