Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DJ5I6L
|
|||
| Drug Name |
SPK-8016
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Haemophilia A [ICD-11: 3B10.0; ICD-9: 286] | Phase 1/2 | [1] | |
| Company |
Spark Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Coagulation factor VIII (F8) | Target Info | Replacement | [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 (NCT03734588) Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors. U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Spark Therapeutics. | |||
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