Drug Information

Drug General Information
Drug ID
D0QF2W
Former ID
DIB002390
Drug Name
AGTC-0106
Synonyms
Alpha-1 antitrypsin deficiency gene therapy, AGTC; RAAV1-CB-hAAT; Gene therapy (alpha-1 antitrypsin deficiency), Applied Genetic Technologies
Indication Alpha-1 antitrypsin deficiency [ICD9: 273.4; ICD10:E88.0] Phase 2 [522924]
Company
Agtc; applied genetic technologies
Target and Pathway
Target(s) Alpha-1-antitrypsin Target Info Modulator [525389]
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
Ref 522924ClinicalTrials.gov (NCT01054339) Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency. U.S. National Institutes of Health.
Ref 525389First gene therapy nears landmark European market authorization. Nat Biotechnol. 2012 Sep;30(9):807-9. doi: 10.1038/nbt0912-807.

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