Drug Information
Drug General Information | Top | |||
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Drug ID |
DBHO19
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Drug Name |
AMT-061
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Synonyms |
etranacogene dezaparvovec
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Drug Type |
Gene therapy
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Indication | Haemophilia B [ICD-11: 3B11.0; ICD-9: 286.1] | Phase 3 | [1] | |
Company |
uniQure
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Target and Pathway | Top | |||
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Target(s) | Coagulation factor IX (F9) | Target Info | . | [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 | |||
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REF 1 | ClinicalTrials.gov (NCT03569891) HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. U.S. National Institutes of Health. | |||
REF 2 | Clinical pipeline report, company report or official report of uniQure. |
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