Drug Information
Drug General Information | Top | |||
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Drug ID |
D0QF2W
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Former ID |
DIB002390
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Drug Name |
AGTC-0106
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Synonyms |
Alpha-1 antitrypsin deficiency gene therapy, AGTC; RAAV1-CB-hAAT; Gene therapy (alpha-1 antitrypsin deficiency), Applied Genetic Technologies
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Indication | Alpha-1 antitrypsin deficiency [ICD-11: 5C5A; ICD-10: E88.0, M79.3; ICD-9: 273.4] | Phase 2 | [1] | |
Company |
Agtc; applied genetic technologies
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Target and Pathway | Top | |||
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Target(s) | Alpha-1-antitrypsin (SERPINA1) | Target Info | Modulator | [2] |
KEGG Pathway | Complement and coagulation cascades | |||
NetPath Pathway | IL6 Signaling Pathway | |||
Panther Pathway | Blood coagulation | |||
Pathway Interaction Database | p73 transcription factor network | |||
FOXA1 transcription factor network | ||||
Reactome | Platelet degranulation | |||
COPII (Coat Protein 2) Mediated Vesicle Transport | ||||
Cargo concentration in the ER | ||||
WikiPathways | Complement and Coagulation Cascades | |||
NRF2 pathway | ||||
Nuclear Receptors Meta-Pathway |
References | Top | |||
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REF 1 | ClinicalTrials.gov (NCT01054339) Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency. U.S. National Institutes of Health. | |||
REF 2 | First gene therapy nears landmark European market authorization. Nat Biotechnol. 2012 Sep;30(9):807-9. |
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